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

FDA Approves Bysanti (milsaperidone) for the treatment of Bipolar I Disorder and Schizophrenia

Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has approved Bysanti (milsaperidone) tablets, a first line therapy for the acute treatment of manic or mixed episodes associated with bipolar I disorder and for the treatment of schizophrenia in adults.

Bysanti is a new chemical entity (NCE) that belongs in the class of atypical antipsychotics. In clinical studies Bysanti demonstrated bioequivalence to iloperidone across the therapeutic dosing spectrum enabling it to leverage well-established knowledge of efficacy and safety derived from a rich clinical development program and more than 100,000 patient-years of real-world experience with Fanapt® (iloperidone). As such Bysanti represents a novel therapeutic option with a trusted safety profile in the treatment of these serious psychiatric conditions.

"The Bysanti approval marks a significant step forward, offering patients and providers a reliable new treatment grounded in extensive clinical heritage," said Mihael H. Polymeropoulos, M.D., President, CEO and Chairman of the Board of Vanda Pharmaceuticals. "Bysanti exemplifies a new era of accelerated innovation in drug development that can transform how we address unmet needs in behavioral health."

Bysanti is currently being tested as a once-daily adjunctive treatment in treatment-resistant major depressive disorder in an ongoing clinical study expected to complete by the end of this year.

Bysanti (milsaperidone), a new chemical entity, rapidly interconverts to iloperidone, providing dual active molecules that work in tandem by antagonizing dopamine D2, serotonin 5-HT2A, and alpha1-adrenergic receptors to modulate key pathways in these disorders. Its safety profile aligns closely with that established for iloperidone.

Bysanti's unique in-class receptor binding profile, featuring strong alpha-adrenergic binding in excess of dopamine and serotonin receptor binding, makes it suitable for further investigation in conditions that include symptoms of hostility, agitation, and hyperarousal.

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

FDA Approves Bysanti (milsaperidone) for the treatment of Bipolar I Disorder and Schizophrenia

FDA Approves Desmoda (desmopressin acetate) Oral Solution for Central Diabetes Insipidus

In continuation of my update on Desmoda

Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the U.S. Food and Drug Administration (FDA) approval of a New Drug Application (NDA) for Desmoda™ (desmopressin acetate) Oral Solution for the management of central diabetes insipidus, also known as arginine vasopressin deficiency (AVP-D), as antidiuretic replacement therapy for patients of all ages.

Desmoda is the first and only FDA-approved desmopressin oral solution

Commercial launch expected on March 9, 2026

Desmoda eliminates tablet splitting and crushing, enabling precise, individualized dosing for patients of all ages

The product is expected to be a significant long-term growth contributor, with potential peak sales of $30-50 million annually and patent protection extending through 2044

Desmoda is the first and only FDA-approved oral liquid formulation of desmopressin, developed to support precise, individualized dosing in a disease where careful titration is essential to maintaining water balance. Desmoda is supplied as a ready-to-use oral solution (0.05 mg/mL) that does not require tablet splitting, crushing, refrigeration, mixing, or shaking.

Eton estimates that there are more than 13,000 patients with central diabetes insipidus in the U.S., including approximately 3,000-4,000 pediatric patients. The company expects product peak sales of $30-50 million annually.

“Desmoda represents one of the most important product launches in Eton’s history. It builds on our strategy of delivering differentiated therapies to rare endocrine patients. By leveraging our existing pediatric endocrinology platform, we believe Desmoda has the potential to become a foundational therapy in this category,” said Sean Brynjelsen, Chief Executive Officer of Eton Pharmaceuticals. “In central diabetes insipidus, effective long-term management depends on accurately matching desmopressin dosing to each patient’s diurnal pattern of water balance. Historically, clinicians have primarily relied on formulations that were not designed for fine dose adjustments or had other administration issues, and both clinicians and families often had to rely on workarounds. Desmoda introduces a liquid solution designed to deliver individualized dosing precision and consistency for patients across the age spectrum.”

Central diabetes insipidus is a rare, but serious condition caused by inadequate production of the hormone vasopressin from the hypothalamus/posterior pituitary. Treatment with desmopressin is the standard of care, but dosing must be individualized to avoid complications related to over- or under-treatment. Oral liquid dosing allows clinicians to incrementally fine-tune therapy and adjust dosing over time as clinical needs evolve.

“Central diabetes insipidus requires careful, individualized management, where dosing precision and flexibility truly matter. Having a liquid formulation of desmopressin has the potential to meaningfully support how we initiate and manage therapy. It’s encouraging to see innovation that directly addresses real-world challenges faced by clinicians and families and gives greater confidence when tailoring treatment across age groups,” said Dr. Lewis Blevins, Director of the California Center for Pituitary Disorders and Professor of Medicine and Neurological Surgery at the University of California, San Francisco.

“For families living with central diabetes insipidus/AVP-D, treatment demands precision, consistency, and careful management of dosing. A liquid option like Desmoda is a meaningful improvement because it helps make sure that the dose is always right, every time. This is very important in cases where the wrong dose can have a big impact," said Muriël Marks, board member of Worldwide Adrenal and Pituitary Organizations (‘WAPO’).

Desmoda will be promoted by Eton’s existing team of pediatric endocrinology rare disease specialists which currently promotes ALKINDI SPRINKLE® (hydrocortisone), KHINDIVITM (hydrocortisone), and INCRELEX® (mecasermin). Desmoda is expected to be available on March 9th exclusively through Anovo, a specialty pharmacy dedicated to serving patients with rare and chronic conditions. Anovo will administer the Eton Cares Program in partnership with Eton Pharmaceuticals. The program provides prescription fulfillment, insurance benefits investigation, educational support, financial assistance for qualified patients, and other services designed to help patients access treatment. Eton Cares will offer co-pay assistance to allow for $0 co-pay for qualifying patients.

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

FDA Approves Desmoda (desmopressin acetate) Oral Solution for Central Diabetes Insipidus

FDA Grants Accelerated Approval to Yuviwel (navepegritide) for Children with Achondroplasia

Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has granted approval under the FDA’s Accelerated Approval Program for Yuviwel (navepegritide; developed as TransCon® CNP), the first and only once-weekly treatment indicated to increase linear growth in children 2 years of age and older with achondroplasia with open epiphyses and the only one to provide continuous systemic exposure to CNP over the weekly dosing interval. Continued approval for this indication, which was based on an improvement in annualized growth velocity (AGV), may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

• The first and only approved achondroplasia therapy to provide continuous systemic exposure to CNP over the weekly dosing interval
• Commercial availability expected during early part of Q2 2026
• Rare Pediatric Disease Priority Review Voucher granted in connection with approval

Achondroplasia is a rare genetic condition causing skeletal dysplasia and, for many affected individuals, an increased risk of muscular, neurological, and cardiorespiratory complications. Yuviwel is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia.

“The approval of once-weekly Yuviwel is a major step forward in the treatment of children with achondroplasia, giving physicians for the first time the option of prescribing a once-weekly medicine backed by compelling efficacy and excellent tolerability data from three randomized, double-blind, placebo-controlled clinical trials,” said Carlos A. Bacino, MD, FACMG, Professor of Molecular and Human Genetics, Baylor College of Medicine and Texas Children’s Hospital. “My goal is to help children and parents develop care plans tailored to their individual needs and objectives, and I look forward to adding Yuviwel to my discussions with them.”

“Little People of America, the largest national advocacy and support organization for people with dwarfism, is committed to ensuring that the voices of people with dwarfism remain central in conversations about research and medical options such as Yuviwel,” said the Board of Directors of Little People of America. “We champion dwarf and disability pride, advocate for inclusion and respect, and foster open dialogue across diverse perspectives. Our goal is to empower individuals and families to make healthcare decisions that reflect their own values and experiences, while pushing for research efforts and new treatment options such as this that could have the potential to support outcomes that truly matter to our community.”

The FDA based its approval of Yuviwel on their review of the clinical package for TransCon CNP submitted with the Company’s New Drug Application, which included safety and efficacy data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data. The pivotal ApproaCH Trial data is available in JAMA Pediatrics.i

“We are confident in Yuviwel’s potential to transform the treatment of achondroplasia and are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. “We have listened to advocacy groups for people with dwarfism to ensure we address what the community actually cares about. This reflects our ongoing commitment to pursue outcomes that patient communities have told us are important to them, and gives the achondroplasia community a new way to look at the promise of pharmacological treatment options.”

Ascendis expects to make Yuviwel available through prescribing physicians in the United States during the early part of the second quarter of 2026. Ascendis plans to offer a suite of patient services for Yuviwel through its U.S. Ascendis Signature Access Program (A.S.A.P.), including support navigating the treatment journey and financial assistance programs for eligible patients.

With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV), which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. This program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.

FDA Grants Accelerated Approval to Yuviwel (navepegritide) for Children with Achondroplasia

FDA Approves Icotyde (icotrokinra) for the Treatment of Plaque Psoriasis

Johnson & Johnson (NYSE: JNJ) announced today that the U.S. Food and Drug Administration (FDA) has approved Icotyde (icotrokinra), an interleukin-23 (IL-23) receptor antagonist for the treatment of moderate-to-severe plaque psoriasis in adults and pediatric patients 12 years of age and older who weigh at least 40 kg who are candidates for systemic therapy or phototherapy. Icotyde is the first and only targeted oral peptide that precisely blocks the IL-23 receptor.

• Johnson & Johnson introduces the first and only IL-23R targeted oral peptide that delivers complete skin clearance and favorable safety profile in a once-daily pill
• Icotyde offers an innovative new option for patients with moderate-to-severe plaque psoriasis to address patients cycling on topical therapies in need of systemic treatment

“Icotyde delivers something unique in psoriasis treatment – combining skin clearance with a favorable safety profile in a once‑daily pill, making it an easy addition to a patient’s routine,” said Linda Stein Gold, M.D., Director of Dermatology Clinical Research at Henry Ford Health.a “With new guidance from the International Psoriasis Council that clarifies when to move beyond cycling on topical treatments to systemic therapy, an innovative option like Icotyde is a potential game‑changer for many adult and adolescent patients.”

Clinical evidence summary

Icotyde met all primary efficacy endpoints and demonstrated a favorable safety profile across four Phase 3 studies including 2,500 patients. The approval is based on an unprecedented body of evidence from the ICONIC clinical development program, which simultaneously evaluated Icotyde in adults and adolescents, high impact sites such as scalp and genital PsO, and in duplicate head-to-head trials versus an active comparator. In the head-to-head superiority studies, approximately 70% of patients achieved clear or almost clear skin (IGA 0/1) and 55% of patients achieved a Psoriasis Area and Severity Index (PASI) 90 response at Week 16.3,b,c Rates of adverse reactions for Icotyde treated patients were within 1.1% of placebo through Week 16 and no new safety signals were identified through Week 52.

“With the FDA approval of Icotyde, Johnson & Johnson is setting a new standard for the treatment of moderate-to-severe plaque psoriasis,” said Jennifer Taubert, Executive Vice President, Worldwide Chairman, Innovative Medicine, Johnson & Johnson. “We’re proud to bring this game-changing innovation to the market, marking a transformative shift in plaque psoriasis management that empowers patients and clinicians to reach their treatment goals.

Unmet need in moderate-to-severe plaque psoriasis

Psoriasis affects more than 8 million Americans, impacting physical comfort and quality of life, especially when lesions are on visible or sensitive areas.5 For many with moderate-to-severe disease, targeted systemic treatments are key. This aligns with International Psoriasis Council guidance to transition to systemic therapy if two cycles of topical medications applied for four weeks fail to bring meaningful improvement.

“Finding the right treatment can take time, during which people with psoriatic disease should be considering multiple factors from efficacy to safety to how the treatment fits into their everyday life,” said Leah M. Howard, J.D., President and CEO of the National Psoriasis Foundation.d “The approval of a novel systemic therapy changes the conversation about treatment options for our community.”

“The approval of Icotyde represents a pivotal moment for people with plaque psoriasis,” said John Reed, M.D., Ph.D., Executive Vice President, R&D, Innovative Medicine, Johnson & Johnson. “At Johnson & Johnson, we are harnessing our scientific expertise to transform cutting-edge science into meaningful solutions for patients. Icotyde is a fundamentally different treatment with the potential to redefine what physicians and patients can expect from psoriasis treatment. 

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

FDA Approves Icotyde (icotrokinra) for the Treatment of Plaque Psoriasis

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

FDA Issues Complete Response Letter for Biohaven's Vyglxia (troriluzole) New Drug Application for Spinocerebellar Ataxia

Biohaven Ltd. (NYSE: BHVN) ("Biohaven"), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases,  announced  it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) seeking approval of Vyglxia (troriluzole) for the treatment of spinocerebellar ataxia (SCA).

 

Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven said, "We are extremely disappointed on behalf of patients by this action from the Office of Neuroscience at FDA. Beyond substantial evidence of safety and efficacy, patients with rare diseases also deserve an efficient, fair and flexible regulatory process that aligns with the urgency of their high unmet medical needs. Such an approach has been mandated by Congress to empower the FDA with maximum regulatory flexibility for rare disease. As a company, we are committed to advancing innovative treatments and remain dedicated to SCA patients despite all the challenges associated with pursuing therapies for rare diseases. Real-world evidence is an important research approach to assessing and delivering new therapies for complex rare diseases but, despite FDA policy initiatives supporting such tools, the front-line review divisions are not yet embracing FDA policy for the use of real-world evidence or the application of regulatory flexibility for rare disease."

Jeremy Schmahmann, M.D., Professor of Neurology at Harvard Medical School and Founding Director of the Ataxia Unit at Massachusetts General Hospital (MGH), added, "Patients with SCA and clinicians who treat them deserve to be heard on this important NDA filing. There is too much at stake for patients. The FDA decision not to listen to disease experts and respect the patient perspective before taking action represents a misstep in the due process, and a failure to deploy regulatory flexibility to evaluate benefit:risk of a medication that has proven to be safe and effective for this rare, debilitating neurodegenerative disease that has no current treatment."

Dr. Coric added, "The development of Vyglxia® (troriluzole) by Biohaven embodies a strong scientific process and deep commitment that is critical to bringing safe and effective treatments to patients with rare diseases like SCA. Our efforts over eight years, included developing the f-SARA scale in collaboration with the FDA and a real-world evidence study in SCA that showed Vyglxia achieved highly consistent, sustained, robust and clinically meaningful treatment effects with a safe, once-daily oral pill that slowed disease progression by 50-70%. The NDA also included data showing Vyglxia reduced the risk of falls and delayed time to becoming wheelchair bound. The leading SCA experts in the United States directly communicated their support of the troriluzole data to the FDA but unfortunately the Office of Neuroscience's inability to collaboratively engage with Biohaven, the patient community and leading experts leave us with concerns about the lack of regulatory flexibility that is being applied for rare, life-threatening conditions. There are a number of common sense solutions and regulatory tools that the Office of Neuroscience could have applied including a fair hearing of the drug's efficacy and safety risks at an Advisory Committee of experts and patients, post-marketing studies, labelling limitations or an accelerated approval pathway. Patients are waiting and the certainty of disease progression for SCA patients far outweighs any residual uncertainty regarding potential design bias or interpretation of study data, especially when the primary outcome measure was achieved in a study protocol and statistical analysis plan that was reviewed by the FDA prior to data analysis. SCA patients deserved approval of Vyglxia and certainly a more balanced interpretation of benefit:risks."

Biohaven remains committed to working with the FDA to find a path forward for its NDA for Vyglxia and plans to meet with the FDA to discuss potential next steps.

Prioritizing Clinical-Stage, Innovative Assets

Biohaven will prioritize resources to focus all its R&D resources on other key programs from its diversified portfolio. Consistent with Biohaven's enduring commitment as a patient-first drug developer, the company's pipeline is focused on a range of disease indications which have limited or no treatment options and are long overdue for therapeutic innovation.

Bruce Car Ph.D., Chief Scientific Officer at Biohaven, commented, "As drug developers we expect setbacks and our diversified portfolio affords us the opportunity to pivot to other key programs. We remain as resilient as ever in following science in order to make a difference in the lives of people with debilitating diseases. Much important work remains, and we are energized and focused on achieving the critical milestones that lie ahead, mindful that days matter and patients are waiting."

Biohaven is initiating strategic portfolio and cost optimization across multiple programs and will focus forward-looking spend on restructuring of business priorities to achieve an approximately 60% reduction in annual direct R&D spend (which excludes personnel and SBC). This may include pausing or delaying non-priority programs to maintain its cash runway to focus on the priority clinical-stage programs over the next year.

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

https://www.drugs.com/nda/vyglxia_251105.html

FDA Approves Arynta (lisdexamfetamine) Oral Solution for ADHD and Binge Eating Disorder

In continuation of my update on lisdexamfetamine

Food and Drug Administration (FDA) has approved Azurity Pharmaceuticals' Arynta™ (lisdexamfetamine) oral solution for the treatment of attention deficit hyperactivity disorder (ADHD) in adults and pediatric patients 6 years and older, and moderate to severe binge eating disorder (BED) in adults.

Arynta is the first oral solution formulation of lisdexamfetamine, providing a new treatment option for patients who have difficulty swallowing or have a preference for using a liquid dosage form.

Lisdexamfetamine is also available as oral capsules and chewable tablets under the brand name Vyvanse® and generic formulations.

Key Clinical Information

Efficacy: The effectiveness of Arynta has been established based on adequate and well-controlled studies of oral lisdexamfetamine dimesylate in treating adults and pediatric patients 6 years and older with ADHD, and adults with moderate to severe BED.

Dosing for ADHD: The recommended starting dosage is 30 mg once daily in the morning for both adults and pediatric patients 6 years and older. Dosage may be adjusted in increments of 10 mg or 20 mg at weekly intervals, up to a maximum dosage of 70 mg once daily.

Dosing for BED: The recommended starting dosage for adults is 30 mg once daily in the morning, titrated in increments of 20 mg at weekly intervals, up to a maximum dosage of 70 mg once daily. Treatment should be discontinued if binge eating does not improve.

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

FDA Approves Arynta (lisdexamfetamine) Oral Solution for ADHD and Binge Eating Disorder

FDA Approves Harliku (nitisinone) for the Treatment of Patients with Alkaptonuria

In continuation of my update on nitisinone

Cycle Pharmaceuticals has announced   the FDA approval of  Harliku (nitisinone) Tablets for the reduction of urine homogentisic acid (HGA) in adult patients with AKU.1

Launching in July 2025, Harliku will be the first and only FDA-approved treatment for AKU,1 an ultra-rare genetic metabolic disorder in which patients have a buildup of HGA that leads to osteoarthritis, ochronosis, and complications in the kidneys, and heart.2 Patients with AKU often develop pain, reduced joint mobility, and require large joint replacements; the symptoms impede their physical functionality, emotional well-being, and quality of life.2,3

The approval of Harliku is based on data from a randomized, no-treatment controlled study of 40 patients with AKU. As part of the intramural research program of the National Human Genome Research Institute at the National Institutes of Health (NIH), Dr. Wendy J. Introne, MD and her team showed that nitisinone helped patients improve pain, energy levels, and physical functioning after three years of treatment, assessed using the 36-Item Short-Form Survey.4

Steve Fuller, Chief Strategy Officer of Cycle Pharmaceuticals commented,

“We are deeply grateful for Cycle’s collaboration with Dr. Wendy Introne, Dr. Bill Gahl, and the broader team at the NIH, whose pioneering work laid the foundation for this FDA approval. We look forward to making Harliku available to U.S. AKU patients as soon as possible and remain committed to supporting the AKU community to the fullest extent of our capabilities.”

“The approval of Harliku is an important advance for the AKU community. Our scientific team has translated decades of research into launching nitisinone as a new treatment option, and we stand hopeful that it can ease the significant burden of AKU,” said Dr. Wendy J. Introne, MD, of NIH’s National Human Genome Research Institute (NHGRI).

Building on the company’s previous success in rare diseases, Harliku will be Cycle Pharmaceuticals’ eighth commercial product in the US.

Indications

Harliku™ is indicated for the reduction of urine homogentisic acid (HGA) in adult patients with alkaptonuria (AKU).

Important Safety Information

Do not prescribe Harliku to patients allergic to nitisinone or any other contained ingredients.

Warnings and Precautions:

Ocular Symptoms and Hyperkeratotic Plaques Due to Elevated Plasma Tyrosine Level:

Ocular signs and symptoms including keratitis, corneal opacities, corneal irritation, corneal ulcers, conjunctivitis, eye pain, and photophobia, have been reported in patients.

Perform slit-lamp examination prior to treatment and regularly thereafter. Reexamine patients if symptoms develop or tyrosine levels are > 500 micromole/L. Assess plasma tyrosine levels in patients with an abrupt change in neurological status.

Perform a clinical laboratory assessment, including plasma tyrosine levels, in patients with an abrupt change in neurological status.

Leukopenia and Severe Thrombocytopenia

In clinical trials, patients with hereditary tyrosinemia type 1 (HT-1) treated with another oral formulation of nitisinone and dietary restriction, developed reversible leukopenia, thrombocytopenia, or both. No patients developed infections or bleeding as a result of the episodes of leukopenia and thrombocytopenia. Monitor platelet and white blood cell counts during Harliku therapy.

Adverse Reactions:

The most common adverse reactions (≥1%) reported in patients with AKU taking nitisinone in clinical trials are elevated tyrosine levels, thrombocytopenia and keratitis.

Drug Interactions:

Nitisinone is a moderate CYP2C9 inhibitor and a weak CYP2E1 inducer. Potential clinical impact of Harliku administration with CYP2C9 substrates. Reduce the dosage of the co-administered drugs metabolized by CYP2C9 by half. Additional dosage adjustments may be needed.

Nitisinone is an inhibitor of OAT1/OAT3. Potential clinical impact of administration with OAT1/OAT3 substrates. Patients should be monitored for p

Ref: https://en.wikipedia.org/wiki/Nitisinone

FDA Approves Harliku (nitisinone) for the Treatment of Patients with Alkaptonuria

FDA Grants Accelerated Approval to Zegfrovy (sunvozertinib) for Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations

Dizal (SSE:688192), a biopharmaceutical company committed to developing novel medicines for the treatment of cancer and immunological diseases, announced  the U.S. Food and Drug Administration (FDA) approval of  Zegfrovy (sunvozertinib) for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations (exon20ins), as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy.

Zegfrovy, which has received Priority Review and Breakthrough Therapy Designation from the FDA, is the only approved targeted oral treatment for NSCLC with EGFR exon20ins. This indication is approved under Accelerated Approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

“We are proud to have developed Zegfrovy, a first-in-class oral therapy that offers a more effective treatment option with enhanced safety and ease of administration for NSCLC patients with EGFR exon20ins,” said Dr. Xiaolin Zhang, CEO of Dizal. “The accelerated approval of Zegfrovy marks a significant milestone that underscores our commitment to developing groundbreaking new medicines for patients with high unmet medical needs around the world.”

Zegfrovy is an oral, irreversible EGFR inhibitor with uniquely designed molecular structure targeting a wide spectrum of EGFR mutations with wild-type EGFR selectivity. In August 2023, Zegfrovy received accelerated approval in China. Today’s FDA approval follows Breakthrough Therapy Designation and Priority Review granted by both the U.S. FDA and the Center for Drug Evaluation (CDE)of China’s National Medical Products Administration (NMPA).

The FDA approval is supported by data from the multinational pivotal study WU-KONG1 Part B (WU-KONG1B), aiming to investigate the efficacy and safety of Zegfrovy in relapsed or refractory NSCLC with EGFR exon20ins. The study results were featured as an oral presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting and were recently published in the Journal of Clinical Oncology.

“As the world's only approved targeted oral therapy for EGFR exon20ins NSCLC, Zegfrovy has expanded the treatment paradigm in this therapeutic area that has long lacked convenient and effective treatment options,” said Pasi A. Jänne, MD, PhD, Dana-Farber Cancer Institute of Harvard Medical School and lead principal investigator of WU-KONG1B. “Research findings from WU-KONG1B have demonstrated Zegfrovy’s significant therapeutic effects with consistent efficacy across both Asian and non-Asian patient populations. Its convenient once-daily oral dosing substantially improves administration convenience and patient adherence, which is an increasingly critical factor as lung cancer care shifts toward chronic disease management. The U.S. approval of Zegfrovy® marks a landmark in scientific advancement and represents a meaningful milestone in addressing the long-standing unmet medical needs of this underserved patient population."

“Zegfrovy has demonstrated breakthrough therapeutic value in the treatment of EGFR exon20ins NSCLC, as shown in a rigorous multinational clinical trial. Its potent antitumor activity, manageable safety profile, and convenient oral administration position it as an optimal treatment option in clinical practice,” said Prof. James Chih-Hsin Yang, MD, PhD, National Taiwan University Cancer Center Hospital and the Co-lead principal investigator of WU-KONG1B. “The approval of Zegfrovy in major global markets not only offers new hope for patients, but also reinforces our commitment to patient-centered research and the continued advancement of precision medicine in lung cancer."

“In NSCLC, EGFR exon20ins represent the third most common type of EGFR mutation. EGFR exon20ins are particularly challenging to treat due to their unique spatial conformation, diverse mutation subtypes, and high heterogeneity. As a result, patients face a poor prognosis and limited treatment options,” said Prof. Mengzhao Wang, MD, PhD, lead principal investigator of the China-based pivotal study WU-KONG6 of Zegfrovy and principal investigator of WU-KONG1B at Peking Union Medical College Hospital, “The results of the WU-KONG6 study demonstrated Zegfrovy’s clinical benefit superior to current options and lead to the drug’s approval in China. The U.S. approval of Zegfrovy will enable more patients around the world to benefit from this drug.”

The FDA simultaneously approved Thermo Fisher Scientific’s Oncomine™ Dx Express Test as a next-generation sequencing (NGS) companion diagnostic (CDx) for Zegfrovy to identify NSCLC patients with EGFR Exon20 insertions. NGS testing is recognized as a critical technology in cancer genomic profiling, facilitating the rapid and precise detection of DNA mutations in tumor cells. Combined with the Ion Torrent™ Genexus™ Dx System, the test delivers NGS results in as little as 24 hours to help inform more timely treatment decisions in patients with EGFR exon20ins NSCLC.

Additionally, Dizal has completed enrollment for its multinational phase III pivotal WU-KONG28 study, evaluating Zegfrovy versus platinum-based doublet chemotherapies in treatment naïve NSCLC patients with EGFR exon20ins across 16 countries and regions. At the 2023 European Society for Medical Oncology (ESMO) Annual Meeting, Dizal reported that Zegfrovy, as a single oral agent, achieved a confirmed objective response rate (ORR) of 78.6% and a median progression-free survival (mPFS) of 12.4 months in the first-line setting. With its potent antitumor activity and favorable safety profile, Zegfrovy demonstrated strong potential as an optimalfirst-line treatment for patients with EGFR exon20ins NSCLC.

Ref : https://www.guidetopharmacology.org/GRAC/LigandDisplayForward?ligandId=11672

FDA Grants Accelerated Approval to Zegfrovy (sunvozertinib) for Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations

FDA Approves Ibtrozi (taletrectinib) for Advanced ROS1-Positive Non-Small Cell Lung Cancer

Nuvation Bio Inc. announced the U.S. Food and Drug Administration (FDA)  approval of  Ibtrozi (taletrectinib) for the treatment of adult patients with locally advanced or metastatic ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC). Ibtrozi is a highly selective, next-generation oral ROS1 tyrosine kinase inhibitor (TKI) designed to address some of the outstanding challenges of treating ROS1+ NSCLC. It has demonstrated high response rates with durable benefit and intracranial activity and is generally well tolerated, providing a new treatment option for patients with advanced ROS1+ NSCLC.

“The FDA approval of Ibtrozi marks a major milestone in the evolution of targeted therapy for advanced ROS1-positive NSCLC,” said David Hung, M.D., Founder, President and Chief Executive Officer of Nuvation Bio. “We believe one of the greatest threats to ROS1-positive lung cancer patients is disease progression, especially in the first-line setting. In pivotal trials, Ibtrozi delivered high response rates with sustained durability—truly meaningful benefits for patients. With its clinically proven efficacy and safety profile, we believe Ibtrozi has the potential to become a new standard for what targeted therapies can achieve in this type of lung cancer. With approvals for Ibtrozi now in the U.S. and China, and additional global filings underway, we remain committed to delivering innovative therapies that help patients stay ahead of their disease.”

ROS1+ NSCLC is a rare and aggressive form of lung cancer, accounting for approximately 2% of new NSCLC cases, or about 3,000 new diagnoses of advanced disease annually in the U.S. The median age at diagnosis for patients with this type of lung cancer is approximately 50 years old, and the disease is more likely to occur in people who have never smoked. Brain metastases are common and a leading cause of disease progression and mortality in this population.

“For people living with advanced ROS1-positive lung cancer, who tend to be diagnosed at a younger age, having another treatment option can make a real difference for them and their loved ones,” said Janet Freeman-Daily, Co-Founder and President of The ROS1ders. “The approval of this new targeted therapy is a meaningful step forward for the advanced ROS1+ lung cancer community and offers hope for patients facing the added challenge of cancer spreading to the brain.”

The FDA approval of Ibtrozi is supported by one of the largest global clinical trial programs in ROS1+ NSCLC to date, with over 300 patients enrolled in the pivotal TRUST-I and TRUST-II studies.

In TRUST-I, Ibtrozi achieved a confirmed overall response rate (cORR) of 90% in TKI-naïve patients. These findings were reinforced by the TRUST-II results, with a cORR of 85% in TKI-naïve patients. The median duration of response (DOR) was not yet reached for either trial, based on a cutoff date that is nearly five months later than that of the pooled TRUST-I and TRUST-II analysis published in April in the Journal of Clinical Oncology. For TRUST-I, with a median follow-up for responses of 40 months, the longest DOR was observed at 46.9 months and ongoing. For TRUST-II, with a median follow-up for responses of 19 months, the longest DOR was observed at 30.4 months and ongoing as of October 2024. Given the single-arm nature of the TRUST clinical studies, median progression-free survival (PFS) is not provided in the label.

Across the pivotal studies, consistent results were also observed among patients who were previously treated with a ROS1 TKI (TKI-pretreated). In TRUST-I, treatment with Ibtrozi achieved a cORR of 52% and median DOR of 13.2 months for TKI-pretreated patients, with median follow-up for responses of 33 months. In TRUST-II, treatment with Ibtrozi achieved a cORR of 62%, and as of October 2024 the median DOR was 19.4 months in these patients, with a median follow-up for responses of 19 months.

Brain metastases are among the most common and devastating complications in advanced ROS1+ NSCLC. Ibtrozi was designed to penetrate the central nervous system (CNS) and has demonstrated consistent intracranial responses in patients with measurable brain metastases at baseline. An intracranial response was achieved in 73% of TKI-naive patients (11/15) and 63% of TKI-pretreated patients (15/24).

“Patients living with advanced ROS1+ non-small cell lung cancer and their healthcare providers are in need of new treatment options,” added Nathan Pennell, M.D., Ph.D., TRUST study investigator and Professor of Medicine at the Cleveland Clinic. “Ibtrozi’s durability of response and ability to effectively penetrate the brain, coupled with a well-characterized and manageable safety profile, further addresses these critical needs for patients. I believe this now-approved therapy offers providers and patients a promising new option for the treatment of advanced ROS1+ non-small cell lung cancer.” Dr. Pennell is a compensated member of Nuvation Bio’s advisory committee.

Ibtrozi was generally well-tolerated, with most adverse events being low grade, transient and manageable. Patients infrequently (7%) discontinued treatment due to treatment-emergent adverse events (TEAEs). The most common adverse reactions (≥20%) included diarrhea (64%), nausea (47%), vomiting (43%), dizziness (22%), rash (22%), constipation (21%), and fatigue (20%). Overall, the majority of CNS events were mild to moderate (~90%) and resolved within days, and dose modifications due to these events were low (~5%). Approximately 90% of reported cases of dizziness were Grade 1 (mild) and transient. Liver enzyme elevations (AST 87%/ALT 85%) and QT prolongation (19%) were manageable with standard monitoring and dose modifications. Ibtrozi is approved as a 600 mg once-daily oral dose, supported by a half-life of approximately 66 hours and broad tissue distribution, including the brain, enabling sustained systemic and CNS exposure.

FDA Approves Ibtrozi (taletrectinib) for Advanced ROS1-Positive Non-Small Cell Lung Cancer

FDA Grants Accelerated Approval for Vanrafia (atrasentan) for Proteinuria Reduction in Primary IgA Nephropathy

Novartis  announced the US Food and Drug Administration (FDA)  accelerated approval for Vanrafia® (atrasentan), a potent and selective endothelin A (ETA) receptor antagonist, for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. This is generally defined as a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g1. Vanrafia is a once-daily, non-steroidal, oral treatment that can be added onto supportive care, including a renin-angiotensin system (RAS) inhibitor with or without a sodium-glucose co-transporter-2 (SGLT2) inhibitor.

Vanrafia was granted accelerated approval based on a prespecified interim analysis of the Phase III ALIGN study measuring the reduction of proteinuria at 36 weeks compared to placebo1. It has not been established whether Vanrafia slows kidney function decline in patients with IgAN. The continued approval of Vanrafia may be contingent upon the verification of clinical benefit from the ongoing Phase III ALIGN study evaluating whether Vanrafia slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline at week 1361. The eGFR data are expected in 2026 and intended to support traditional FDA approval.

“Today’s approval marks an important milestone for people living with IgA nephropathy, offering a new option that can be seamlessly integrated into their existing treatment plan, with no REMS requirement,” said Richard Lafayette, M.D., F.A.C.P., Professor of Medicine, Nephrology and Director of the Glomerular Disease Center at Stanford University Medical Center, and Vanrafia ALIGN Study Investigator and Steering Committee Member. “Vanrafia is a selective ETA receptor antagonist that effectively reduces proteinuria, a major risk factor in IgAN. Taking early, decisive action is critical to help improve outcomes for these patients who too often progress toward kidney failure.”

IgAN is a progressive, rare kidney disease in which the immune system attacks the kidneys, often causing glomerular inflammation and proteinuria10. With almost 13 out of every million people in the US diagnosed per year, it is one of the most common autoimmune kidney diseases, and each person’s journey is unique11,12. Up to 50% of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring maintenance dialysis and/or kidney transplantation3-10, and response to treatment can vary12,13. Effective, targeted therapies with different mechanisms of action can help physicians select the most appropriate treatment for patients.

REF: https://en.wikipedia.org/wiki/Atrasentan#:~:text=in%20April%202025.-,Names,under%20the%20brand%20name%20Vanrafia.

FDA Approves Mezofy (aripiprazole) Oral Film for the Treatment of Schizophrenia

In continuation of my update on Aripiprazole

CMG Pharmaceuticals, an affiliate of Cha Biotech, announced on the 16th that it had received product approval for the schizophrenia treatment drug Mezofy (formerly Depibzo) from the U.S. Food and Drug Administration (FDA).

Mezofy is an oral film-type schizophrenia treatment drug (ingredient name: aripiprazole) developed by CMG Pharmaceuticals. Patients with mental illnesses including schizophrenia often refuse or spit out medication, but the film-type drug can be taken without water and easily dissolves in the mouth, solving this problem.

CMG Pharmaceuticals first applied for FDA approval in December 2019. At that time, CMG Pharmaceuticals was the world’s first film-type schizophrenia treatment company to knock on the FDA’s door. However, the supplementary inspection was delayed due to problems with overseas raw material factories and the impact of the COVID-19 pandemic.

Meanwhile, Opipza, aripiprazole film treatment from China's LP Pharma, received FDA approval in July of last year. CMG Pharmaceuticals then reapplied for product approval in October of last year and received marketing approval.

Mezofy is the fourth product from a domestic pharmaceutical company to receive FDA approval for an improved new drug. An improved new drug is not simply a copy of a new drug whose patent has expired, but rather an improvement in the dosage method by changing the efficacy or formulation. Unlike simple generic drugs, it is protected separately by a patent.

The company explained, “The price of improved new drugs is set higher than that of generic drugs, and they can be marketed and prescribed under the product name rather than the ingredient name, which is advantageous in increasing awareness in the market.”

CMG Pharmaceuticals expects Mezofy to be launched in the US in the first half of 2026. According to Data Monitor, a US market research and analysis firm, the US schizophrenia treatment market is the largest in the world, at 12 trillion won.

The company predicted that the market for Mezofy would expand to over KRW 22 trillion when its indications are expanded to include bipolar disorder, major depressive disorder, autism spectrum disorder, and Tourette's disorder.

The company said, “We plan to complete the selection of a local distribution partner in the U.S. by the second half of this year,” and “We will work with our distribution partners to establish a competitive drug pricing strategy.”

CMG Pharmaceuticals has set a goal of achieving annual sales of over 100 billion won within five years of entering the U.S. market. CMG Pharmaceuticals CEO Lee Ju-hyung said, “After proving the excellence of Mezofy in the U.S. market, we will advance into other overseas markets.”

At the time of the initial application for product approval, the product name was Depipzo. The company explained that in collaboration with the global pharmaceutical branding specialist Brand Institute, it decided on Mezofy as a name that is easier to remember and less likely to cause prescription errors.

Ref: https://en.wikipedia.org/wiki/Aripiprazole

FDA Approves Atzumi (dihydroergotamine) Nasal Powder for the Acute Treatment of Migraine

Shin Nippon Biomedical Laboratories, Ltd. (TSE:2395),  announced  the U.S. Food and Drug Administration (FDA) approval of    New Drug Application (NDA) for Atzumi™(dihydroergotamine (DHE)) nasal powder for the acute treatment of migraine with or without aura in adults. Atzumi was previously known as STS101.

 

Migraine is a neurological disorder that is thought to be the result of temporary changes in the chemicals, nerves and blood vessels in the brain, with symptoms that are often incapacitating. According to the American Migraine Foundation, approximately 40 million Americans live with migraine. It is the second leading cause of disability worldwide in terms of time lost to disability and most common cause of disability among young women.

"The approval of Atzumi is a milestone to celebrate, providing a new option for the acute treatment of migraine combining long-proven benefits of DHE with a patient-friendly and easy-to-use delivery system developed based on SNBL's novel intranasal drug delivery platform technology," said Dr. Ryoichi Nagata, President and CEO of Satsuma. "We believe that Atzumi will contribute to improving the quality of life of patients struggling for relief from these highly disabling problems."

"DHE plays a unique clinical role in the acute treatment of migraine, providing patients long lasting effects and the unique ability to provide benefit even when taken late in a migraine attack. The convenience of Atzumi, the only DHE nasal powder, will offer patients ease of use combined with the important known DHE clinical advantages", said Dr. Stewart J. Tepper, M.D., Vice President of the New England Institute for Neurology and Headache in Stamford, Connecticut.

About Atzumi

Atzumi is a proprietary drug device product incorporating both Satsuma's advanced nasal powder formulation of dihydroergotamine (DHE) administered via its unique nasal delivery device. The product is designed to provide patients an easy-to-use and easy-to-carry treatment option.

The FDA approval for Atzumi is based on two clinical studies (Phase 1 PK trial and ASCEND Phase 3 open-label, long-term safety trial), which demonstrated fast absorption, rapid achievement of high DHE plasma concentrations, and sustained DHE plasma levels over time as well as safety and tolerability in subjects with migraine.

About Dihydroergotamine (DHE)

Since its approval in 1946, DHE has long been recommended in published migraine treatment guidelines as a first-line acute treatment option for migraine and has significant advantages versus other anti-migraine treatments for many patients. However, disadvantages of current DHE liquid nasal spray and injectable products, including invasive and burdensome administration and/or sub-optimal clinical performance, have limited the widespread use of DHE.

Ref: https://en.wikipedia.org/wiki/Dihydroergotamine

https://pubchem.ncbi.nlm.nih.gov/compound/Dihydroergotamine#section=2D-Structure