Aldeyra Therapeutics Receives Complete Response Letter from the U.S. Food and Drug Administration for the Reproxalap New Drug Application for the Treatment of Signs and Symptoms of Dry Eye Disease

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to discovering and developing innovative therapies designed to treat immune-mediated diseases, announced receipt of a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) of reproxalap, an investigational drug candidate, for the treatment of dry eye disease. The CRL stated that there is “a lack of substantial evidence consisting of adequate and well-controlled investigations … that the drug product will have the effect it purports or is represented to have under the conditions of use prescribed, recommended, or suggested in its proposed labeling” and that “the application has failed to demonstrate efficacy in adequate and well controlled studies in the treatment of signs and symptoms of dry eye disease.” The letter also stated that the “inconsistency of study results raises serious concerns about the reliability and meaningfulness of the positive findings” and that the “totality of evidence from the completed clinical trials does not support the effectiveness of the product.” Consistent with prior NDA reviews of reproxalap, no safety or manufacturing concerns were identified.

During the NDA review, label drafts were provided by the FDA in December 2025 and again in March 2026. Aldeyra does not believe that label negotiations were completed.

The FDA recommended that the reasons for failure in certain trials be explored, and that populations or certain conditions in which reproxalap may be effective be identified. The FDA did not recommend conducting additional trials or request submission of additional confirmatory evidence. As such, Aldeyra does not currently expect to pursue additional clinical trials, and intends to expeditiously request a Type A meeting to understand the actions needed for NDA approval. Per Prescription Drug User Fee Act (PDUFA) goals, the target Type A meeting date is within 30 days of receipt of the meeting request.

“To the thousands of American and Canadian patients who participated in our clinical trials and to the tens of millions of patients with dry eye disease worldwide, I want to assure you that we will work with urgency to support the FDA in enabling market access to what is, to our knowledge, the only drug with clinical activity within minutes of administration in patients with dry eye disease, a condition that is today treated with medications that require weeks or months of treatment to achieve even modest improvement,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra.

https://go.drugbank.com/drugs/DB16688

Nanomedicine offers targeted solutions for breast cancer treatment

Breast cancer (BCA) is one of the most common cancers worldwide, with high mortality and morbidity in women. This review focuses on the applications of nanotechnology, nanomaterials (NMs), and nanoparticles (NPs) in BCA diagnosis and therapy. Nanotechnologies, nanocarriers, and nano-encapsulation versus conventional counterparts are discussed. Various drug formulations into lipid NPs, nanoemulsions, polymeric NPs, and metal-based NPs enhance bioavailability and therapeutic efficacy, overcoming limitations of conventional formulations. Clinical specialists have achieved improved outcomes in BCA detection and monitoring using nanotechnology, ultimately improving patients' quality of life.

Introduction

Cancer is a leading cause of death globally. Breast cancer accounts for 30% of all cancer cases and 15% of cancer‑related deaths in women. The PI3K/AKT/mTOR signaling pathway plays a crucial role in BCA development and progression. Nanomedicine applies NMs and NPs for prevention, diagnosis, and treatment. This review addresses challenges of conventional therapies (lack of target specificity, drug resistance, systemic toxicity) and highlights how nanotechnology overcomes these limitations.

General Aspects of NMs and NPs

NMs have at least one dimension in 1–100 nm and a large surface‑to‑volume ratio, conferring novel properties. Reducing particle size increases solubility and surface interactions. Nanotechnology improves pharmacokinetics, enables targeted delivery, enhances permeability and retention effects in tumors, and reduces required drug doses.

Different Aspects of BCA

Statistics: GLOBOCAN 2022 reported 2,296,840 new BCA cases (ASIR 46.8 per 10⁵) and 666,103 deaths (ASMR 12.7 per 10⁵) worldwide.

Molecular Subtypes: BCA is classified by hormone receptor and HER2 status: Luminal A (~40%, ER⁺/PR⁺, HER2⁻), Luminal B (~20%, ER⁺/PR⁺, HER2⁺/⁻), HER2‑enriched (~10‑15%, ER⁻/PR⁻, HER2⁺), and triple‑negative breast cancer (TNBCA, ~15‑20%, ER⁻/PR⁻/HER2⁻). TNBCA is aggressive, occurs in younger women, has high recurrence and metastasis rates, and lacks targetable proteins.

Challenges: Treatment resistance, recurrence, adverse effects, low cellular absorption, and multidrug resistance.

Therapies: Surgery, chemotherapy, radiotherapy, hormonal therapy, and immunotherapy. Nanotechnology offers innovative carriers to overcome limitations.

Carriers and Nanocarriers for Drug Delivery

Conventional carriers have limited tumor response and affect normal cells. Nanocarriers include lipid nanoparticles (LNPs), nanoemulsions (NEs), polymeric NMs, and metallic NPs. They enhance drug stability, absorption, encapsulation efficiency, bioavailability, and controlled release. NEs improve oral delivery of poorly soluble drugs and reduce toxicity.

Nanocarriers for BCA

Chitosan‑based nanocarriers exploit electrostatic interactions with cancer cells, enhance cellular uptake, and open tight junctions. Quaternary ammonium chitosan improves penetration. Chitosan NPs deliver genes, drugs, and natural compounds; induce phototherapy‑mediated tumor ablation; and support combination therapy.

Clinical results: Nanocarriers improved drug delivery and outcomes. Photothermal nanomaterials (PTT) with nanotechnology enhanced metastatic BCA treatment, reduced damage to healthy cells, and synergized with chemotherapy/immunotherapy. Cyclophosphamide NEs in rats showed remarkable tumor reduction. Exemestane‑loaded polymer‑lipid hybrid nanoparticles (PLH NPs) improved oral bioavailability (>3.5‑fold) and tumor inhibition (62% vs. 31% for conventional suspension) in mice.

Major Metallic Nanocarriers

• Gold (Au) NPs: Biocompatible, easy surface modification, effective against TNBCA via Rad6 conjugation inducing mitochondrial dysfunction. Clinical translation limited by toxicity in liver, kidneys, spleen.

• Silver (Ag) NPs: High photon attenuation; ethyl cellulose‑coated Ag NPs inhibited TNF‑α in BCA cells.

• Copper (Cu) NPs: Bioactive; 5‑fluorouracil loaded into β‑cyclodextrin‑Cu NPs showed sustained release and anticancer activity against TNBCA.

• Iron oxide (Fe₃O₄) NPs: Magnetic core‑shell NPs (Fe₃O₄‑poly(N‑isopropylacrylamide)‑grafted chitosan) delivered methotrexate with 94% entrapment efficiency; enhanced antitumor activity against MCF‑7 cells at 40°C and pH 5.5.

REF: https://www.xiahepublishing.com/2996-3427/OnA-2025-00027

FDA Approves Komzifti (ziftomenib) for the Treatment of Adults with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

Kura Oncology, Inc. (Nasdaq: KURA) and Kyowa Kirin Co., Ltd. (TSE: 4151)  announced the U.S. Food and Drug Administration (FDA)   approval of Komzifti™ (ziftomenib) for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options. Komzifti is the first and only once-daily, oral menin inhibitor approved for R/R NPM1-mutated (NPM1-m) AML, a devastating blood cancer with limited treatment options.

“Komzifti combines compelling efficacy, a favorable safety profile, compatibility with concomitant medications, and convenient once-daily oral administration in a population with few effective treatment options. These features highlight Komzifti’s potential to serve as the menin inhibitor of choice in its approved indication,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “Together with our partner, Kyowa Kirin, we remain committed to advancing development of Komzifti across the treatment continuum for AML, where its best-in-class profile offers potential for even greater impact in combination regimens and earlier lines of therapy. We are fully prepared to launch Komzifti today and deliver this new medicine to patients in need.”

NPM1 mutations are among the most common founder mutations in AML, occurring in approximately 30% of cases. Historically, approximately 20% of patients with NPM1-m AML do not respond to front-line therapy. Of those who do respond, 70% will relapse within 3 years, most within 12 months. Early relapse and declining survival with each recurrence underscore the urgent need for treatment approaches that deliver lasting remission.

Approval is supported by the pivotal KOMET-001 trial (NCT04067336), which evaluated Komzifti’s safety and efficacy in 112 R/R NPM1-m AML patients. The rate of complete remission (CR) plus CR with partial hematologic recovery (CRh) was 21.4% (95% CI: 14.2, 30.2). The median duration of CR+CRh was 5.0 months (95% CI: 1.9, 8.1) and the median time to first response in patients who achieved a CR or CRh was 2.7 months (range: 0.9 to 15 months). 88% of patients who achieved CR or CRh did so within 6 months of initiating Komzifti. These data from the Prescribing Information are generally consistent with findings recently published in the Journal of Clinical Oncology.1

“Komzifti addresses a critical need for adult patients with R/R NPM1-m AML, many of whom are older and unable to tolerate intensive chemotherapy or transplant,” said Eunice Wang, M.D., Chief of the Leukemia Service and Professor of Oncology at Roswell Park Comprehensive Cancer Center. “The clinical data demonstrate deep and durable responses with a manageable safety profile, including no drug-drug interactions and no Boxed Warnings for QTc prolongation or Torsades de Pointes – key advantages for patients on multiple concurrent medications. This approval equips physicians with a new oral therapy to integrate into care and improve outcomes for this vulnerable patient population.”

The most common adverse reactions (≥20%), including laboratory abnormalities, were aspartate aminotransferase increased, infection without an identified pathogen, potassium decreased, albumin decreased, alanine aminotransferase increased, sodium decreased, creatinine increased, alkaline phosphatase increased, hemorrhage, diarrhea, nausea, fatigue, edema, bacterial infection, musculoskeletal pain, bilirubin increased, potassium increased, differentiation syndrome, pruritus, febrile neutropenia, and transaminases increased. Komzifti includes a Boxed Warning for differentiation syndrome, a well-studied mechanism-based risk in drugs that restore differentiation. Absence of clinically meaningful drug-drug interactions can ease the use of Komzifti with concomitant therapies, including those that cause QTc interval prolongation. QTc interval prolongation was ≤ Grade 3 in 12% of patients and no Grade 4 or Grade 5 QTc interval prolongation was reported. QTc interval prolongation of any cause occurred in 10% of the 70 patients 65 years of age or older.

“The approval of Komzifti underscores our commitment to advancing precision medicines to address the genetic drivers of disease in hematology and oncology,” said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. “In AML, where many patients face severe disease progression and limited treatment options, the evolution toward targeted therapies such as Komzifti represents a major step forward and offers potential to transform existing standards of care. We are proud to partner with Kura Oncology in bringing this important therapy to patients and their families.”

In November 2024, Kura Oncology and Kyowa Kirin entered into a global strategic collaboration to develop and commercialize Komzifti. The collaboration builds on Kyowa Kirin’s leadership and expertise in hematologic malignancies. Under the terms of the collaboration, Kura leads development, regulatory and commercial strategy in the U.S. and is responsible for manufacturing Komzifti. The companies will jointly perform certain commercialization activities in accordance with a co-created U.S. territory commercialization plan. Outside the U.S., Kyowa Kirin leads development, regulatory and commercial strategy and is responsible for commercializing Komzifti.

As part of its commitment to helping patients access Komzifti, Kura has established a support program, Kura RxKonnect™, to minimize barriers to access and reimbursement for appropriate patients prescribed Komzifti. Kura RxKonnect is available 8:00 am – 8:00 pm ET by phone at (844) KuraPSP (844-587-2777) or online at www.KuraRxKonnect.com.

https://en.wikipedia.org/wiki/Ziftomenib

FDA Approves Komzifti (ziftomenib) for the Treatment of Adults with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

FDA Approves Myqorzo (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy

Cytokinetics, Incorporated (Nasdaq: CYTK) announced the U.S. Food and Drug Administration (FDA) approval of  Myqorzo™ (aficamten, 5 mg, 10 mg, 15 mg, 20 mg tablets for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. Myqorzo is an allosteric and reversible inhibitor of cardiac myosin motor activity. In patients with oHCM, myosin inhibition with Myqorzo reduces cardiac contractility and left ventricular outflow tract (LVOT) obstruction.

“This is a historic moment for our company and for the patients we serve, as we fulfill our promise to translate our science into medicines that may make a meaningful difference in patients’ lives,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “Our first FDA approval stands as a testament to the strength of our science and the bold, trailblazing research that has defined Cytokinetics’ leadership in muscle biology. I’m pleased that the approved label and REMS reflect the distinct characteristics of Myqorzo including a straightforward, flexible dosing regimen, no requirement for drug-drug interaction monitoring and a predictable safety profile. I am profoundly grateful for the many years of passion and persistence shown by patients with obstructive HCM, as well as healthcare professionals, advocates, partners and employees who have contributed so importantly to reaching this key milestone.”

The full U.S. Prescribing Information for Myqorzo includes a Boxed WARNING for the risk of heart failure. Myqorzo reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction. Echocardiogram assessments are required prior to and during treatment with Myqorzo to monitor for systolic dysfunction. Initiation of Myqorzo in patients LVEF <55% is not recommended. Decrease the dose of Myqorzo if LVEF <50% and ≥40%. Interrupt the dose of Myqorzo if LVEF <40% or if the patient experiences heart failure symptoms or worsening clinical status due to systolic dysfunction. Because of the risk of heart failure due to systolic dysfunction, Myqorzo is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Myqorzo REMS Program. Please see additional Important Safety Information including Boxed WARNING below.

Myqorzo is expected to be available in the U.S. in the second half of January 2026. Cytokinetics will support patients with Myqorzo & You™, a personalized program for patients prescribed Myqorzo in the U.S. to help navigate the treatment journey, provide disease and product education, and offer support with insurance benefits investigations or financial assistance for those eligible. For more information, call 833-Myqorzo (833-697-6796).

“HCM is a heart muscle disease associated with a significant symptom burden. This approval of a new drug, Myqorzo, represents a meaningful addition to the treatment options available for symptomatic obstructive HCM patients,” said Martin Maron, M.D., Director, Hypertrophic Cardiomyopathy Center, Lahey Hospital and Medical Center, and Principal Investigator of SEQUOIA-HCM. “In SEQUOIA-HCM, Myqorzo improved exercise capacity and reduced symptoms while also being well-tolerated. For these reasons, Myqorzo represents an important step forward in how we care for people living with obstructive HCM.”

https://en.wikipedia.org/wiki/Aficamten

FDA Approves Myqorzo (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy

FDA Approves Adquey (difamilast 1%) Ointment for the Treatment of Mild-to-Moderate Atopic Dermatitis

Acrotech Biopharma Inc., a bio-pharmaceutical company focused on developing and commercializing innovative propriety medications, in collaboration with Otsuka Pharmaceutical Co., Ltd.,  announced  the U.S. Food and Drug Administration (FDA) approval of  New Drug Application (NDA) for Adquey (difamilast 1%) ointment, for the topical treatment of mild-to-moderate atopic dermatitis (AD) in adults and pediatric patients aged 2 and older.

Adquey is a novel, non-steroidal, topical phosphodiesterase 4 (PDE4) inhibitor. This approval provides a new non-steroidal, topical treatment option for the millions of Americans living with atopic dermatitis. "The approval of Adquey represents a significant milestone in our commitment to advancing dermatology care," said Ashish Anvekar, President of Acrotech Biopharma Inc. "Patients and clinicians have long sought effective, long-term, non-steroidal treatments that can manage both the inflammation and pruritus associated with eczema. We intend to bring this valuable treatment option to the patients in the U.S. as soon as possible."

Clinical Trial Results

FDA approval was supported by multiple studies including pivotal Phase III controlled trials, which demonstrated that a significantly greater proportion of patients treated with Adquey achieved Investigator’s Global Assessment (IGA) success compared to vehicle (placebo) after four weeks of treatment. The safety profile of Adquey AD was consistent across all three trials. The most common adverse reactions (≥1% and greater than vehicle) was nasopharyngitis. Less common (<1%) adverse reactions in subjects treated with Adquey included application site folliculitis, contact dermatitis, application site rash, and molluscum contagiosum.

https://en.wikipedia.org/wiki/Difamilast

FDA Approves Adquey (difamilast 1%) Ointment for the Treatment of Mild-to-Moderate Atopic Dermatitis

Elevar Therapeutics Announces FDA Acceptance for Review of New Drug Application for Lirafugratinib as Second-line Cholangiocarcinoma Treatment

Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd. and a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, today announced that the U.S. Food and Drug Administration (FDA) has completed its filing review of the New Drug Application (NDA) for lirafugratinib, an investigational therapy, for the treatment of patients with cholangiocarcinoma (CCA) with FGFR2 fusions or rearrangements who have received prior therapy. The FDA determined that the NDA is sufficiently complete to permit a substantive review and granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of September 27, 2026.

• With Priority Review designation, FDA set a Sept. 27 PDUFA date for approval decision
• Lirafugratinib achieved a 46.5% ORR, in CCA patients with FGFR2 fusion and rearrangement

Priority Review designations are given to applications for medicines that, if approved, would lead to “significant improvements in the safety or effectiveness of the treatment” of a serious condition, according to the FDA. The Priority Review designation of the lirafugratinib NDA was supported by positive clinical data from the Phase 1/2 ReFocus trial (NCT04526106), which demonstrated a confirmed objective response rate (ORR) of 46.5% in the patients with the proposed indication. Its safety profile in the clinical data has been shown to be predictable and manageable through dose adjustments.

“Lirafugratinib has established a compelling clinical profile that differentiates it from existing treatment options,” said Dong-Gun Kim, chief executive officer of Elevar. “We are very pleased with the FDA’s priority review designation and focused on advancing the review process efficiently to bring this therapy to patients as quickly as possible.”

CCA, also known as bile duct cancer, is rare, with about 8,000 people in the U.S. diagnosed each year, according to the American Cancer Society.

Elevar Therapeutics continues to evaluate lirafugratinib in ongoing clinical development programs, including studies in other FGFR2-altered solid tumors. Any future indications will be subject to regulatory review and approval.

For more information about lirafugratinib, visit ElevarTX.com.

About Lirafugratinib

Lirafugratinib (RLY-4008) is a potent, selective, and oral small molecule inhibitor of FGFR2, a receptor tyrosine kinase that is frequently altered in certain cancers. FGFR2 is one of four members of the FGFR family, a set of closely related proteins with highly similar protein sequences and properties. Preclinically, lirafugratinib demonstrated FGFR2-dependent killing in cancer cell lines and induced regression in in vivo models with minimal inhibition of other targets, including other members of the FGFR family. In addition, lirafugratinib demonstrated strong activity against known clinical on-target resistance mutations in vitro and in vivo preclinical models. Lirafugratinib is currently being evaluated in a clinical trial to enroll additional patients with previously treated, advanced or metastatic solid tumors other than CCA harboring FGFR2 fusion or rearrangement, who have not been treated with prior FGFR inhibitors.

Elevar Therapeutics Announces FDA Acceptance for Review of New Drug Application for Lirafugratinib as Second-line Cholangiocarcinoma Treatment

Eylea HD (aflibercept) Approved by FDA for the Treatment of Macular Edema Following Retinal Vein Occlusion (RVO) and for Monthly Dosing Across Approved Indications

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) announced the U.S. Food and Drug Administration (FDA) approval of Eylea HD (aflibercept) Injection 8 mg for the treatment of patients with macular edema following retinal vein occlusion (RVO) with up to every 8-week dosing after an initial monthly dosing period. The FDA also approved an every 4-week (monthly) dosing option for some patients who may benefit from resuming this dosing schedule across approved indications: wet age-related macular degeneration (wAMD), diabetic macular edema (DME), diabetic retinopathy (DR) and RVO. 

Aflibercept is a combination of fluorouracil, leucovorin, and irinotecan,

flurouracil 

Folinic acid,

Irinotecan

"We believe these approvals further position Eylea HD as a treatment of choice for certain retinal diseases and underscore our relentless commitment to meeting the needs of patients and the retina specialists who treat them,” said George D. Yancopoulos, M.D., Ph.D., co-Founder, Board Co-Chair, President and Chief Scientific Officer, at Regeneron. “Eylea HD is the first treatment for retinal vein occlusion that can potentially cut the number of injections that patients receive in half compared to existing therapies. And with the addition of a monthly dosing option for all four approved Eylea HD indications, physicians now have greater flexibility and optionality to tailor treatment to meet individual patient needs.”

The FDA approval for the treatment of RVO is based on data from the Phase 3 QUASAR trial that evaluated the efficacy and safety of Eylea HD compared to Eylea® (aflibercept) Injection 2 mg in patients with RVO. QUASAR met its primary endpoint at 36 weeks, with Eylea HD patients dosed every 8 weeks (after either 3 or 5 monthly doses) achieving non-inferior visual acuity gains compared to those receiving Eylea dosed every 4 weeks. The Eylea HD results were consistent across patients with branch retinal vein occlusions, and those with central retinal or hemiretinal vein occlusions. In RVO, the most common adverse reactions reported in ≥3% of patients treated with Eylea HD were intraocular pressure increased, vision blurred, cataract, conjunctival hemorrhage, ocular discomfort/eye pain/eye irritation and vitreous detachment.

In regard to the Eylea HD pre-filled syringe (PFS), Regeneron continues to coordinate with Catalent Indiana, LLC (part of Novo Nordisk A/S) as it works to resolve the outstanding issues identified from a July 2025 FDA general site inspection (not specific to Eylea HD). As previously disclosed, Regeneron also plans to submit to the FDA an application to include an alternate PFS manufacturing filler for the Eylea HD BLA by January 2026.

Eylea HD is approved with dosing intervals from every 8 to 16 weeks for patients with wAMD and DME (following 3 initial monthly doses), every 8 to 12 weeks for patients with DR (following 3 initial monthly doses), and every 8 weeks for patients with RVO (following 3 to 5 initial monthly doses). In clinical trials, some Eylea HD patients did not maintain a response with extended dosing intervals after successful response to initial monthly doses; these patients may benefit from resuming every 4-week dosing.

https://en.wikipedia.org/wiki/Aflibercept#:~:text=Aflibercept%20is%20a%20recombinant%20fusion%20protein%20consisting%20of%20vascular%20endothelial,of%20the%20human%20IgG1%20immunoglobulin.

https://en.wikipedia.org/wiki/Fluorouracil

https://en.wikipedia.org/wiki/Folinic_acid

https://en.wikipedia.org/wiki/Irinotecan

Eylea HD (aflibercept) Approved by FDA for the Treatment of Macular Edema Following Retinal Vein Occlusion (RVO) and for Monthly Dosing Across Approved Indications

Achieve Life Sciences Announces FDA Acceptance of Cytisinicline New Drug Application for Treatment of Nicotine Dependence for Smoking Cessation

Achieve Life Sciences, Inc. (Nasdaq: ACHV), a late-stage specialty pharmaceutical company focused on the global development and commercialization of cytisinicline for treatment of nicotine dependence, today announced that the U.S. Food and Drug Administration (FDA) has accepted the cytisinicline New Drug Application (NDA) for a new treatment for smoking cessation in adults. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) targeted action date of June 20, 2026.

“The FDA’s acceptance of our NDA validates the totality of our product development program,” said Dr. Cindy Jacobs, President and Chief Medical Officer of Achieve Life Sciences. “Our application is supported by a decade of rigorous research and comprehensive data from thousands of participants. We've built compelling scientific and clinical results as a foundation that, if approved, positions cytisinicline to potentially address a significant medical need. We’re eager to engage constructively with the FDA as we progress through the NDA review process.”

The company’s NDA details a comprehensive clinical development program, with more than 2,000 clinical trial participants contributing to the body of evidence. The ORCA-2 and ORCA-3 Phase 3 trials showed cytisinicline administered for either 6 or 12 weeks, alongside standard behavioral support, demonstrated significantly greater smoking abstinence rates by the end of treatment and in long-term abstinence through week 24 as compared to placebo. In addition, safety data will include over 400 participants who have received at least six months of cumulative cytisinicline exposure and over 200 participants receiving at least 1 year of cumulative cytisinicline exposure, with no new safety concerns as reported by the Data Safety Monitoring Committee.

“Smoking is the leading cause of preventable death and disease, claiming the lives of nearly half a million Americans each year, and costing the American economy more than an estimated $600 billion a year,” said Rick Stewart, Chief Executive Officer of Achieve Life Sciences. “The FDA's acceptance of our NDA filing underscores nicotine dependence as an important public health need that demands action. We're energized by this milestone and remain laser-focused on our mission, working toward commercial readiness for the second half of 2026, pending FDA approval. Each year, an estimated 15 million Americans attempt to quit smoking, and we're committed to providing those who are ready to quit a new tool so they can break free from nicotine dependence.”

https://en.wikipedia.org/wiki/Cytisine

Achieve Life Sciences Announces FDA Acceptance of Cytisinicline New Drug Application for Treatment of Nicotine Dependence for Smoking Cessation

FDA Approves Kygevvi (doxecitine and doxribtimine) for the Treatment of Thymidine Kinase 2 Deficiency

UCB (Euronext Brussels: UCB), a global biopharmaceutical company, today announced that Kygevvi has been granted approval by the U.S. Food and Drug Administration (FDA) for the treatment of adults and pediatric patients living with thymidine kinase 2 deficiency (TK2d), with an age of symptom onset on or before 12 years.1 It is the first and only approved treatment for these patients living with TK2d.

TK2d is an ultra

Doxecitine/doxribtimine

-rare, life-threatening, genetic mitochondrial disease characterized by progressive (worsening over time) and severe muscle weakness (myopathy) with no approved treatment options beyond supportive care until now.2,3,4,5 It is often fatal, with those experiencing initial symptoms on or before the age of 12 years facing a high risk of premature death (often occurring within 3 years after symptom onset).6 It is estimated that the worldwide prevalence of TK2d is 1.64 [0.5, 3.1] cases per 1,000,000 people.7

“The approval of doxecitine and doxribtimine represents a pivotal moment for the TK2d community who previously had no FDA-approved treatment options for this rare genetic mitochondrial disease beyond supportive [palliative] care,” said Donatello Crocetta, Chief Medical Officer at UCB. “We extend heartfelt thanks to the patients, families and friends, advocates, healthcare providers and dedicated clinical trial teams who have partnered with us on this important journey.”

“It’s hard to overstate the importance of this FDA approval for those diagnosed with TK2d. This is an ultra-rare disease community in dire need of treatment options. For too long, caregivers and their families have had to endure the burden of this disease," said Kristen Clifford, United Mitochondrial Disease Foundation President and CEO. “Having the first-ever FDA-approved therapy for TK2d in this patient population not only meets a critical medical need - it represents something greater - hope for the future."

“I’ve been studying mitochondrial diseases for more than three decades and have witnessed firsthand the impact TK2d has on patients and their families. We have been waiting for an approved treatment for many years, and this approval marks a significant milestone in how we can support and manage this debilitating condition,” said Dr. Michio Hirano, Professor of Neurology and Chief of the Division of Neuromuscular Medicine at Columbia University Irving Medical Center.

Supporting data

The Kygevvi approval is supported by safety and efficacy data from one Phase 2 clinical study, two retrospective chart review studies, and an expanded access use program*.1,8,9,10,11 These studies included a total of 82 unique patients treated with Kygevvi or pyrimidine nucleosides with an age of TK2d symptom onset ≤12 years. Efficacy was assessed by comparing overall survival in these pediatric and adult treated patients to an external control group of untreated patients who were matched to treated patients using age of TK2d symptom onset (≤ 2 years or >2 to ≤ 12 years).1 A total of 78 matched pairs were identified. The results showed that survival time from treatment start was improved; treatment reduced the overall risk of death from treatment start by approximately 86% (95% CI: 61%, 96%).1 Of the 78 treated patients included in the survival analysis, the median age of TK2d symptom onset was 1.5 years (range: 0.01 to 12 years).1 The median duration of treatment was 4 years (range: 1 day to 12 years) and the median dose received was 762 mg/kg/day (range: 260 to 800 mg/kg/day).1

The most common adverse reactions (incidence ≥5%) are diarrhea, abdominal pain (including abdominal pain upper), vomiting, alanine aminotransferase increased (ALT), and aspartate aminotransferase increased (AST).1

A regulatory review of doxecitine and doxribtimine is currently underway by the EMA (European Medicines Agency), and further regulatory submissions are planned. Kygevvi is currently not approved for use in any indication by any regulatory authority outside of the U.S. UCB expects Kygevvi to be commercially available in the U.S. in Q1, 2026. To further its mission of equitable care, UCB will provide a personalized support program for Kygevvi that places the needs of patients and caregivers at the forefront.

In the U.S., Kygevvi received Orphan Drug, Breakthrough, Priority Review and Rare Pediatric Disease designations from the FDA.12,13 With this approval by FDA, UCB was awarded a Rare Pediatric Disease Priority Review Voucher (RPDPRV) redeemable for a priority review for a future marketing application.

About Kygevvi

Kygevvi is a combination of doxecitine and doxribtimine, both pyrimidine nucleosides, indicated for the treatment of thymidine kinase 2 deficiency (TK2d) in adults and pediatric patients with an age of symptom onset on or before 12 years.1 Administration of Kygevvi is intended to incorporate the pyrimidine nucleosides, deoxycytidine and deoxythymidine, into skeletal muscle mitochondrial DNA.1 This action restores mitochondrial DNA copy number in TK2d mutant mice.1

Important safety information for Kygevvi1

Increase in Liver Transaminases

Elevated liver transaminase [alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST)] levels were reported in patients treated with Kygevvi. Obtain baseline liver transaminase (ALT, AST) and total bilirubin levels in patients prior to treatment initiation with Kygevvi. If signs or symptoms consistent with liver injury are observed, interrupt treatment with Kygevvi until liver transaminase (ALT, AST) and total bilirubin levels have either returned to baseline or stabilized at a new baseline value. Consider permanently discontinuing Kygevvi if signs or symptoms consistent with liver injury persist or worsen. Monitor liver transaminases and total bilirubin levels yearly and as clinically indicated.

Gastrointestinal Adverse Reactions

Diarrhea and vomiting leading to hospitalization, dose reduction, and permanent discontinuation were reported in patients treated with Kygevvi. Based on the severity of the diarrhea and/or vomiting, reduce the dosage of Kygevvi or interrupt treatment until diarrhea and/or vomiting improves or returns to baseline. Consider restarting Kygevvi at the last tolerated dose, and increase the dose as tolerated. For persistent or recurring diarrhea and/or vomiting, consider discontinuing Kygevvi permanently and provide supportive care with electrolyte repletion as clinically indicated.

https://en.wikipedia.org/wiki/Doxecitine/doxribtimine

FDA Approves Kygevvi (doxecitine and doxribtimine) for the Treatment of Thymidine Kinase 2 Deficiency

FDA Approves Contepo (fosfomycin) for Injection for the Treatment of Complicated Urinary Tract Infections

The U.S. Food and Drug Administration (FDA) has approved Contepo (fosfomycin) for injection for the treatment of patients 18 years of age and older with complicated urinary tract infections (cUTI), including acute pyelonephritis, caused by susceptible isolates of Escherichia coli and Klebsiella pneumoniae.

Contepo is an epoxide antibacterial that works by disrupting bacterial cell wall synthesis through covalently binding and inhibiting phosphoenolpyruvate transferase (MurA), thereby blocking the synthesis of peptidoglycan. The drug is bactericidal against Enterobacterales.

Dosing and Administration

The recommended dosage of Contepo is 6 grams administered every 8 hours by intravenous infusion over 1 hour in patients 18 years of age or older with an estimated creatinine clearance greater than 50 mL/min. The duration of therapy is up to 14 days and should be guided by the severity of infection and the patient's clinical status. Dosage adjustments are required for patients with renal impairment.

Clinical Trial Results

The FDA approval was based on data from a multinational, double-blind clinical trial (Trial 1) that enrolled 464 adults hospitalized with cUTI, including acute pyelonephritis. The study compared Contepo 6 grams intravenously every 8 hours to piperacillin/tazobactam 4.5 grams intravenously every 8 hours for 7 days of therapy, with treatment allowed for up to 14 days in bacteremic patients.

In the microbiological modified intent-to-treat (mMITT) population of 339 patients, Contepo demonstrated efficacy with regard to overall response at the test-of-cure visit. Overall success, defined as clinical cure plus microbiological eradication, was achieved in 63.5% (108/170) of Contepo-treated patients compared to 55.6% (94/169) of piperacillin/tazobactam-treated patients.

Important Safety Information

The most common adverse reactions (incidence ≥2%) observed in clinical trials were transaminase elevations, hypokalemia, neutropenia, nausea, vomiting, diarrhea, hypocalcemia, hypernatremia, headache, and hypophosphatemia.

Contepo contains 1,980 mg of sodium in each vial. The high sodium load associated with its use may result in changes in serum electrolytes, such as increased levels of serum sodium and decreased levels of potassium, calcium, and phosphorus. A low-sodium diet is recommended during Contepo treatment, and healthcare providers should monitor serum electrolyte levels and fluid status during treatment.

Contepo has been shown to prolong the QT interval in some patients and should be avoided in patients with known QT prolongation or ventricular arrhythmias, including a history of torsade de pointes. Healthcare providers should monitor electrolytes during treatment with Contepo.

Contepo is contraindicated in patients with known serious hypersensitivity to fosfomycin or any of the excipients.

FDA Approves Contepo (fosfomycin) for Injection for the Treatment of Complicated Urinary Tract Infections

FDA Approves Inlexzo (gemcitabine intravesical system) for the Treatment of Non-Muscle Invasive Bladder Cancer

In continuation of my update on gemcitabine 

Johnson & Johnson (NYSE:JNJ) announced  the U.S. Food and Drug Administration (FDA) approval of  Inlexzo (gemcitabine intravesical system), a new, potentially practice-changing approach for treating patients with certain types of bladder cancer, addressing the need for additional options following unsuccessful BCG therapy and for patients refusing or ineligible for bladder removal surgery (radical cystectomy). Inlexzo, previously referred to as TAR-200, is indicated for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors.1

Inlexzo is designed for patients seeking bladder preservation and is the first and only intravesical drug releasing system (iDRS) to provide extended local delivery of a cancer medication into the bladder. Inlexzo remains in the bladder for three weeks per treatment cycle for up to 14 cycles.1 A healthcare professional places Inlexzo into the bladder using a co-packaged urinary catheter and stylet to insert it into the bladder. Inlexzo is placed in an outpatient setting in a few minutes, without the need for general anesthesia or further monitoring immediately post-insertion within the healthcare provider’s office.1

“When we acquired this novel therapy in 2019, our ambition was to give patients with bladder cancer a renewed sense of hope and belief,” said Jennifer Taubert, Executive Vice President, Worldwide Chairman, Innovative Medicine, Johnson & Johnson. “In an area that has seen little progress for more than 40 years, Inlexzo delivers a first-of-its-kind breakthrough innovation with a bright future ahead.”

The approval is supported by data from the SunRISe-1 ( NCT04640623) single arm, open-label Phase 2b clinical study.1 Results show 82 percent of patients with BCG-unresponsive NMIBC treated with Inlexzo achieved a complete response (CR), meaning no signs of cancer were found after treatment (95 percent confidence interval [CI], 72, 90).1 This high response rate demonstrated strong durability, and 51 percent of these patients maintained a complete response for at least one year.1

In the SunRISe-1 clinical trial supporting this approval, the most common adverse reactions (≥15 percent) including laboratory abnormalities, were urinary frequency, urinary tract infection, dysuria, micturition urgency, decreased hemoglobin, increased lipase, urinary tract pain, decreased lymphocytes, hematuria, increased creatinine, increased potassium, increased aspartate aminotransferase (AST), decreased sodium, bladder irritation, and increased alanine transaminase (ALT).1

“I see many patients that ultimately become BCG-unresponsive and often face life-altering bladder removal. These patients now may be ideal candidates for newly approved Inlexzo,” said Sia Daneshmand, M.D., TAR-200 SunRISe-1 principal investigator, and Professor of Urology, Director of Urologic Oncology at the Norris Comprehensive Cancer Center, Keck School of Medicine of University of Southern California.* “In my experience, Inlexzo is well-tolerated and delivers clinically meaningful results. This will change the way we treat appropriate patients that haven’t responded to traditional therapy.”

“We are proud of the science that has brought us to this historic moment,” said John Reed, M.D., Ph.D., Executive Vice President, R&D, Innovative Medicine, Johnson & Johnson. “Inlexzo is a novel therapy with powerful efficacy and demonstrated safety profile. As the only major healthcare company that hosts both pharmaceuticals and medical devices, we leveraged the speed and scale of Johnson & Johnson to accelerate innovation and deliver this important therapy to patients.”

“At BCAN, our mission has always been to advocate for better todays and more tomorrows for everyone impacted by bladder cancer. This approval represents the kind of progress that brings new options to a community that urgently needs them,” said Meri-Margaret Deoudes, CEO, Bladder Cancer Advocacy Network (BCAN).** “Patients with bladder cancer need guidance and collaboration with providers to navigate bladder-sparing treatment options, including newly approved treatments like Inlexzo, so they can move forward feeling well-informed and confident.”

Leading to today’s approval, the FDA granted Inlexzo Breakthrough Therapy Designation (BTD), Real-Time Oncology Review (RTOR), and Priority Review.

Johnson & Johnson is committed to helping patients access our treatments. Once a patient and their doctor have decided that Inlexzo is right for the patient, J&J withMe provides a simple, comprehensive patient support program offering cost support, a dedicated Care Navigator and educational resources, at no cost to the patient.

About SunRISe-1, Cohort 2SunRISe-1 ( NCT04640623), Cohort 2, was a single arm, open-label Phase 2b clinical study that evaluated the safety and efficacy of Inlexzo monotherapy for BCG-unresponsive NMIBC patients with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for, or elected not to undergo, radical cystectomy. The primary endpoint for Cohort 2 was complete response (CR) rate at any time point, and secondary endpoints include duration of response (DOR).

About Non-Muscle Invasive Bladder Cancer (NMIBC) and the Current Standard of Care Non-muscle invasive bladder cancer (NMIBC) is a type of non-invasive bladder cancer that can be classified as low, intermediate, or high risk depending on the presence of characteristics including tumor size, presence of multiple tumors, and carcinoma in situ (CIS).2 NMIBC with CIS makes up approximately 10 percent of patients with NMIBC.3 The current standard of care for NMIBC is Bacillus Calmette-Guérin (BCG), which is a weakened form of the bacteria found in tuberculosis treatment. Though effective, some patients become unresponsive to it and may experience challenges.4,5 Radical cystectomy is currently recommended for NMIBC patients who fail BCG therapy; it is a life-altering surgery with a high degree of morbidity and adverse impact on life, and has a post-surgery mortality rate of three to eight percent.6,7 Given that NMIBC typically affects older patients, many may be unwilling or unfit to undergo radical cystectomy.

About Inlexzo

Inlexzo is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. Inlexzo is an intravesical system enabling extended release of gemcitabine into the bladder. It is placed in a few minutes without general anesthesia or further monitoring immediately post-insertion within the healthcare provider’s office.

The safety and efficacy of Inlexzo is being evaluated in clinical trials in patients with MIBC in SunRISe-4, and NMIBC in SunRISe-1, SunRISe-3, and SunRISe-

https://en.wikipedia.org/wiki/Gemcitabine#:~:text=More%20than%2010%25%20of%20users,back%20pain%2C%20and%20muscle%20pain.

FDA Approves Inlexzo (gemcitabine intravesical system) for the Treatment of Non-Muscle Invasive Bladder Cancer

FDA Approves Tonmya (cyclobenzaprine hydrochloride sublingual tablets) for the Treatment of Fibromyalgia

Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP),  announced the U.S. Food and Drug Administration (FDA) approval of  Tonmya™ (cyclobenzaprine HCl sublingual tablets) for the treatment of fibromyalgia in adults. Tonmya is a first-in-class, non-opioid, once-daily bedtime analgesic with a unique sublingual (under the tongue) formulation that is designed for rapid absorption into the bloodstream. Tonmya is the first new FDA-approved therapy for the treatment of fibromyalgia in over 15 years.

“The FDA approval of Tonmya as a first-line treatment for fibromyalgia represents a landmark advancement for the millions of people in the U.S. suffering from the debilitating pain this condition causes,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “At Tonix, we recognized the transformative potential of pursuing a new approach with Tonmya for fibromyalgia, a chronic overlapping pain condition (COPC), that has gone without innovation for many years. We are hopeful that effectively treating pain with Tonmya could help improve the lives of people with this chronic syndrome.”

“The chronic pain of fibromyalgia is debilitating to every aspect of a person’s life, including causing sleep disturbance and fatigue, all of which can negatively impact someone’s ability to carry out their daily activities,” said Sharon Waldrop, a person with lived experience and founder of the Fibromyalgia Association. “For over 15 years, this community has been underserved and waiting for new treatment options. This approval is a promising step forward and brings renewed hope to millions.”

The approval incorporated efficacy from two double-blind, randomized, placebo-controlled, Phase 3 clinical trials of nearly 1,000 patients in total that evaluated Tonmya as a bedtime treatment for fibromyalgia. Across both Phase 3 trials, Tonmya significantly reduced daily pain scores compared to placebo at 14 weeks, the primary endpoint. Additionally, a greater percentage of study participants taking Tonmya experienced a clinically meaningful (≥30%) improvement in their pain after three months, compared to placebo.

Across three Phase 3 clinical trials with over 1,400 patients evaluated, Tonmya was generally well tolerated. The most common adverse events (incidence ≥2% and at a higher incidence in Tonmya-treated patients compared to placebo-treated patients) included oral hypoesthesia (numbness in the mouth), oral discomfort, abnormal product taste, somnolence (drowsiness), oral paresthesia (tingling, pricking or burning in the mouth), oral pain, fatigue, dry mouth, and aphthous ulcer (canker sore).

“For many years, rheumatologists like myself and other healthcare professionals have had to manage fibromyalgia with limited options that do not adequately meet treatment needs for the majority of patients,” said Philip Mease, M.D., Director of Rheumatology Research at the Providence Swedish Medical Center and Clinical Professor at the University of Washington School of Medicine. “Tonmya is a novel treatment approach that targets nonrestorative sleep that is characteristic of fibromyalgia and can impact core symptoms, specifically pain.”

The latest Phase 3 trial, RESILIENT, was recently published in Pain Medicine with data on primary and secondary endpoints measuring pain, patient’s global impression of change, patient-reported symptoms and function, sleep disturbance, and fatigue.

“I know firsthand how the chronic pain of fibromyalgia significantly disrupts my patients’ lives.” Andrea L. Chadwick, M.D., MSc, FASA, Anesthesiology, Pain, and Perioperative Medicine at The University of Kansas Health System. “Treatments that are processed through the liver can result in metabolites that could affect a medicine’s efficacy and safety over time. Tonmya is administered sublingually which is designed to reduce pain quickly and durably with a tolerable safety profile.”

Tonix thanks the participants and investigators involved in its fibromyalgia clinical trials, and FDA for its commitment to approving new treatments for this condition.

Tonmya is expected to be available for adult patients in the U.S. with fibromyalgia beginning in the fourth quarter of this year.

https://en.wikipedia.org/wiki/Cyclobenzaprine

FDA Approves Tonmya (cyclobenzaprine hydrochloride sublingual tablets) for the Treatment of Fibromyalgia

FDA Approves Brinsupri (brensocatib) for the Treatment of Non-Cystic Fibrosis Bronchiectasis

Insmed Incorporated (Nasdaq: INSM),  announced  the U.S. Food and Drug Administration (FDA)  approval of  first-in-class Brinsupri (brensocatib 10 mg and 25 mg tablets), an oral, once-daily treatment for non-cystic fibrosis bronchiectasis (NCFB) in adults and children 12 years and older. Brinsupri is the first and only FDA-approved treatment for NCFB, giving hundreds of thousands of patients and clinicians across the U.S. an option to manage this chronic and progressive disease that can lead to permanent lung damage and lung function decline.

"This FDA approval represents a potential paradigm shift in how we approach non-cystic fibrosis bronchiectasis," said Doreen Addrizzo-Harris, M.D., FCCP, the Fiona and Stanley Druckenmiller Professor of Pulmonary, Critical Care and Sleep Medicine at NYU Grossman School of Medicine and Director of the NYU Langone Health Bronchiectasis and NTM Program, and ASPEN investigator. "For the first time, we have a treatment that directly targets neutrophilic inflammation and addresses a root cause of bronchiectasis exacerbations. Based on the strength of the data and the impact we've seen in patients, I believe this could become the new standard in non-cystic fibrosis bronchiectasis care."

There are approximately 500,000 people in the U.S. diagnosed with NCFB, and it is estimated that millions more are living with this disease globally. Unlike other respiratory diseases that are characterized by airway narrowing, bronchiectasis causes airways to permanently widen, making it harder to clear mucus and bacteria, leading to persistent inflammation and infection. A hallmark of bronchiectasis is frequent exacerbations, or flares, when symptoms worsen, such as coughing, increased mucus, shortness of breath and fatigue.

"Non-cystic fibrosis bronchiectasis deeply affects the lives of people living with this chronic lung condition, impacting both their physical health and emotional well-being," added Elisha Malanga, Executive Director of the Bronchiectasis and NTM Association. "Many patients experience frequent flares, which can disrupt daily life and potentially lead to disease progression. The FDA approval of brensocatib represents a significant and long-awaited advancement as the first approved therapy for non-cystic fibrosis bronchiectasis. Our hope is that treatments like this will enable people with bronchiectasis to manage their condition."

This approval is based on data from the Phase 3 ASPEN and Phase 2 WILLOW studies, which were both published in the New England Journal of Medicine. In ASPEN, patients taking Brinsupri 10 mg or 25 mg had a 21.1% and 19.4% reduction in annual rate of exacerbations respectively, as compared to placebo. Both dosage strengths of Brinsupri also met several exacerbation-related secondary endpoints, including significantly prolonging the time to first exacerbation and significantly increasing the proportion of patients remaining exacerbation-free over the treatment period. Patients who received Brinsupri 25 mg experienced statistically significant less decline in lung function, as measured by forced expiratory volume in one second (FEV₁) after using a bronchodilator, at week 52. The safety of Brinsupri was also evaluated in both studies. The most common adverse reactions ≥2% in the ASPEN trial included upper respiratory tract infection, headache, rash, dry skin, hyperkeratosis, and hypertension. The safety profile for adult patients with NCFB in WILLOW was generally similar to ASPEN, except for a higher incidence of gingival and periodontal adverse reactions in WILLOW.

"The FDA approval of the first-ever treatment for non-cystic fibrosis bronchiectasis is a historic milestone for patients and for Insmed," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. "By keeping patients at the center of everything we do, we have once again delivered a first-in-class medicine for a disease with no prior approved treatments. This is an incredible achievement in medicine. We're deeply grateful to the patients, providers, and advocates who made this possible – this is just the beginning of what we can accomplish together for this community."

Brinsupri is a first-in-class dipeptidyl peptidase 1 (DPP1) inhibitor, designed to inhibit the activation of enzymes (neutrophil serine proteases) in neutrophils that are key drivers of chronic airway inflammation in NCFB. Brinsupri is the first approved therapy to address the underlying inflammatory process of NCFB.

In parallel, applications for brensocatib with the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) have been accepted, and the Company plans to file in Japan in 2025. Commercial launches are anticipated in 2026, pending approval in each territory.

Brinsupri is now available in the U.S. by prescription through a comprehensive specialty pharmacy network.

https://en.wikipedia.org/wiki/Brensocatib

FDA Approves Brinsupri (brensocatib) for the Treatment of Non-Cystic Fibrosis Bronchiectasis

FDA Approves Sephience (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced   U.S. Food and Drug Administration (FDA)  approval of Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU.

"We are excited to have reached this important milestone for those affected by PKU," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The broad labeling reflects the potential of Sephience to meet the significant unmet need of PKU patients. The Sephience clinical data along with our expertise in launching rare disease therapies position Sephience to become the future standard of care. Our experienced customer facing teams are ready to bring this therapy to children and adults with PKU in the United States as quickly as possible."

The FDA approval is based on the evidence of significant efficacy and safety from the

Phase 3 APHENITY trial as well as durability of treatment effect in the APHENITY long-term extension study.

"The approval marks an exciting milestone for the PKU community," said Catherine Warren, Executive Director of the National PKU Alliance. "This progress brings renewed hope, and we are eager to see the positive impact this new treatment option will have on advancing care and potentially improving quality of life for individuals of all ages and PKU subtypes that respond to this therapy."

Sephience was recently granted marketing authorization by the European Commission. Review of approval applications is ongoing in several other countries including Japan and Brazil.

About Sephience (sepiapterin)

Sephience is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients with phenylketonuria (PKU). Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Through its mechanism of action, Sephience is able to effectively reduce blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients. Sephience is approved in the European Economic Area and the United States.

Indication and Important Safety Information

Indication

Sephience is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). Sephience is to be used in conjunction with a phenylalanine (Phe)-restricted diet.

Contraindications

None

Important Safety Information

Treatment with Sephience should be directed by physicians knowledgeable in the management of PKU. Biochemical response to Sephience can only be determined by a therapeutic trial with careful monitoring of ongoing dietary and nutritional balance to ensure adequate Phe control.

Warnings and Precautions

Increased Bleeding: Sephience may increase the risk of bleeding. Bleeding events, including superficial hematomas, prolonged bleeding, and heavy menstrual bleeding have occurred in patients treated with Sephience. Inform patients about the risk of bleeding associated with Sephience and have patients follow up with their healthcare provider should such a bleeding event occur. Consider treatment interruption with Sephience in patients with active bleeding.

Hypophenylalaninemia: Some pediatric patients receiving Sephience experienced hypophenylalaninemia. Monitor blood Phe levels during treatment and modify the dosage of Sephience and/or dietary protein and Phe intake as needed to ensure adequate blood Phe level control. Frequent blood monitoring is recommended in the pediatric population.

Interaction with Levodopa: In a 10-year post-marketing safety surveillance program for a non-PKU indication using another drug that is a phenylalanine hydroxylase (PAH) activator, 3 patients with underlying neurological disorders experienced seizures, exacerbation of seizures, over-stimulation, and irritability during co-administration with levodopa. Monitor patients who are receiving levodopa for changes in neurological status during treatment with Sephience.

Adverse Reactions

Most common adverse reactions with Sephience (≥2% and > placebo) were diarrhea, headache, abdominal pain, hypophenylalaninemia, feces discoloration, and oropharyngeal pain.

Drug Interactions

Avoid concomitant use of drugs known to inhibit folate synthesis dihydrofolate reductase (DHFR) (e.g., trimethoprim, methotrexate, trimetrexate, pemetrexed, pralatrexate, raltitrexed, and piritrexim) while taking Sephience. Concomitant administration of such drugs may reduce sepiapterin metabolism to BH4. If concomitant use is not avoidable, monitor blood Phe levels.

Sephience and PDE-5 inhibitors (e.g., sildenafil, vardenafil, or tadalafil) induce vasorelaxation and may reduce blood pressure. Monitor for signs and symptoms of hypotension.

For medical information, product complaints, or to report an adverse event, please call 1–866–562–4620 or email at usmedinfo@ptcbio.com.

https://en.wikipedia.org/wiki/Sepiapterin

FDA Approves Sephience (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria