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 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

FDA Approves Pivya (pivmecillinam) for the Treatment of Uncomplicated Urinary Tract Infections

Today, the U.S. Food and Drug Administration approved Pivya (pivmecillinam) tablets for the treatment of female adults with uncomplicated urinary tract infections (UTIs) caused by susceptible isolates of Escherichia coli, Proteus mirabilis and Staphylococcus saprophyticus.

“Uncomplicated UTIs are a very common condition impacting women and one of the most frequent reasons for antibiotic use,” said Peter Kim, M.D., M.S., director of the Division of Anti-Infectives in the FDA’s Center for Drug Evaluation and Research. “The FDA is committed to fostering new antibiotic availability when they prove to be safe and effective, and Pivya will provide an additional treatment option for uncomplicated UTIs.”

Uncomplicated UTIs are bacterial infections of the bladder in females with no structural abnormalities of their urinary tract. Approximately one-half of all women experience at least one UTI in their lifetime.

Pivya’s efficacy in treating females 18 years of age or older with uncomplicated UTIs was assessed in three controlled clinical trials comparing different Pivya dosing regimens to placebo, to another oral antibacterial drug and to ibuprofen (an anti-inflammatory drug). The primary measure of efficacy for the three trials was the composite response rate, which included clinical cure (resolution of the symptoms of the uncomplicated UTI that were present in patients at trial entry and no new symptoms) and microbiological response (demonstration that the bacteria cultured from patients’ urine at trial entry was reduced). The composite response rate was assessed approximately 8 to 14 days after patients were enrolled into the studies. In the clinical trial comparing Pivya to placebo, 62% of the 137 subjects who received Pivya achieved the composite response compared to 10% of the 134 who received placebo. In the clinical trial comparing Pivya to another oral antibacterial drug, 72% of the 127 subjects who received Pivya achieved composite response compared to 76% of the 132 who received the comparator drug. In the clinical trial comparing Pivya to ibuprofen, 66% of the 105 subjects who received Pivya achieved composite response compared to 22% of the 119 who received ibuprofen.

The most common side effects of Pivya included nausea and diarrhea.

Patients should not use Pivya if they have a known history of severe hypersensitivity to Pivya or other beta-lactam antibacterial drugs. Patients should also not use Pivya if they have primary or secondary carnitine deficiency resulting from inherited disorders of mitochondrial fatty acid oxidation and carnitine metabolism, or if they are suffering from porphyria.

Pivya comes with certain warnings and precautions such as hypersensitivity reactions, severe cutaneous adverse reactions, carnitine depletion, Clostridioides difficile-associated diarrhea and interference with a newborn screening test for isovaleric acidemia, a rare metabolic disorder.

Pivya was granted Priority Review and Qualified Infectious Disease Product designations for this indication.

The FDA granted the approval of Pivya to UTILITY therapeutics Ltd.

FDA Approves Pivya (pivmecillinam) for the Treatment of Uncomplicated Urinary Tract Infections

FDA Approves Fruzaqla (fruquintinib) for Previously Treated Metastatic Colorectal Cancer

Takeda announced the U.S. Food and Drug Administration (FDA) has approval of  Fruzaqla™ (fruquintinib), an oral targeted therapy for adults with metastatic colorectal cancer (mCRC) who have been previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if RAS wild-type and medically appropriate, an anti-EGFR therapy. Fruzaqla is the first and only selective inhibitor of all three VEGF receptor kinases approved in the U.S. for previously treated mCRC regardless of biomarker status.1,2 This approval was received under Priority Review more than 20 days ahead of the scheduled PDUFA date of November 30, 2023.

"There is a pressing need for new treatments for individuals with metastatic colorectal cancer, who have had limited options and continue to face poor outcomes. Fruzaqla is the first novel chemotherapy-free treatment option approved for patients in the U.S. regardless of biomarker status in more than a decade,” said Teresa Bitetti, president of the Global Oncology Business Unit at Takeda. “For far too long, healthcare providers and patients have had limited options when selecting a therapy for metastatic colorectal cancer. Fruzaqla has the potential to offer a significant survival benefit to patients without negatively impacting their quality of life.”

The approval of Fruzaqla is based on data from two large Phase 3 trials: the multi-regional FRESCO-2 trial, data from which were published in The Lancet, along with the FRESCO trial conducted in China, data from which were published in JAMA. The trials investigated Fruzaqla plus best supportive care versus placebo plus best supportive care in patients with previously treated mCRC. Both FRESCO and FRESCO-2 met their primary and key secondary efficacy endpoints and showed consistent benefit among a total of 734 patients treated with Fruzaqla. Safety profiles were consistent across trials.

“Patients with metastatic disease are often fragile and fatigued – due to both their condition as well as the therapies they have been exposed to. An oral, chemotherapy-free option that offers a survival benefit despite treatment with prior therapies is a critical need for treating metastatic colorectal cancer,” said Cathy Eng, M.D., FACP, at Vanderbilt University Medical Center. “Colorectal cancer is a highly heterogeneous disease, making it difficult to bring advancements to patients whose cancer has metastasized. I look forward to being able to offer a new solution to appropriate patients.”

In the United States, approximately 153,000 new cases of CRC will be diagnosed in 2023, representing 7.8% of all new cancer cases.3,4 Approximately 70% of patients with CRC will experience metastatic disease, whether at diagnosis or after treatment. Metastases are the main cause of CRC-related mortality.

“We have witnessed firsthand the physical and emotional toll metastatic colorectal cancer has on patients, their families and their care teams,” said Michael Sapienza, Chief Executive Officer, at Colorectal Cancer Alliance. “We are encouraged to see the continued progress in providing new options to patients.”

Ref ; https://en.wikipedia.org/wiki/Fruquintinib

FDA Approves Fruzaqla (fruquintinib) for Previously Treated Metastatic Colorectal Cancer

FDA Approves Aurlumyn (iloprost) as the First Medication to Treat Severe Frostbite

The U.S. Food and Drug Administration approved Aurlumyn (iloprost) injection to treat severe frostbite in adults to reduce the risk of finger or toe amputation.

"This approval provides patients with the first-ever treatment option for severe frostbite,” said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiology and Nephrology in the FDA’s Center for Drug Evaluation and Research. “Having this new option provides physicians with a tool that will help prevent the lifechanging amputation of one’s frostbitten fingers or toes."

Frostbite can occur in several stages, ranging from mild frostbite that does not require medical intervention and does not cause permanent skin damage, to severe frostbite when both the skin and underlying tissue are frozen and blood flow is stopped, sometimes requiring amputation. Iloprost, the active ingredient in Aurlumyn, is a vasodilator (a drug that opens blood vessels) and prevents blood from clotting.

Iloprost’s efficacy in treating severe frostbite was primarily established in an open-label, controlled trial that randomized 47 adults with severe frostbite, who all received aspirin by vein and standard of care, into one of three treatment groups. One of these groups (Group 1) received iloprost by vein (intravenously) for 6 hours daily for up to 8 days. The two other groups received other medications that are unapproved for frostbite, given with iloprost (Group 2) or without iloprost (Group 3). The primary measure of efficacy was a bone scan obtained 7 days after initial frostbite that was used to predict the need for amputation of at least one finger or toe.

On day 7, the bone scan finding predictive of needing amputation was observed in 0% (0 of 16) patients receiving iloprost alone (Group 1) compared to 19% (3 of 16) patients in Group 2 and 60% (9 of 15) patients in Group 3. The presence of the bone scan abnormality was significantly lower in the two groups receiving iloprost. Most patients had follow-up information on whether they subsequently underwent at least one finger or toe amputation. The need for amputation was consistent with the bone scan findings.

The most common side effects of Aurlumyn include headache, flushing, heart palpitations, fast heart rate, nausea, vomiting, dizziness, and hypotension (blood pressure that is too low). Aurlumyn also has a warning and precaution noting that it may cause symptomatic hypotension.

Aurlumyn received Priority Review and Orphan Drug designations for this indication.

Iloprost was originally approved in 2004 for the treatment of pulmonary arterial hypertension.  The FDA granted the approval of Aurlumyn to Eicos Sciences Inc.

Ref; https://en.wikipedia.org/wiki/Iloprost.

FDA Approves Aurlumyn (iloprost) as the First Medication to Treat Severe Frostbite

FDA Approves Lampit (nifurtimox) for the Treatment of Chagas Disease in Children

Bayer announced  that  the United States Food and Drug Administration (FDA) has approved Lampit (nifurtimox) for use in pediatric patients (from birth to less than 18 years of age and weighing at least 2.5 kg) for the treatment of Chagas disease (American Trypanosomiasis) caused by Trypanosoma cruzi (T. cruzi).1 Lampit, an antiprotozoal medication will be available in a new, dividable tablet that can be split on the scored lines by hand.1-3 According to prescribing instructions, the tablet is specially formulated to disperse in water, which can aid in the dosing and administration to pediatric patients who may have difficulty swallowing whole or half tablets. 

This indication is approved under accelerated approval based on the number of treated patients who became immunoglobulin G (IgG) antibody negative or who showed an at least 20% decrease in optical density on two different IgG antibody tests against antigens of T. cruzi. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Chagas is an infectious tropical disease that affects an estimated 300,000 people in the U.S.4 The disease is endemic throughout much of Latin America, though it is a growing health concern in the U.S.5,6 Approval of a treatment for pediatric patients is an important milestone.

“Chagas disease can strike at any age, and early detection and treatment are important. This is especially relevant for children,” said Aleksandra Vlajnic, MD, senior vice president and head of Medical Affairs for the Americas at Bayer. “The importance of treating children is a major reason behind Bayer’s collaboration with health authorities to enhance access to Lampit as a means to provide treatment for Chagas disease.”

The FDA approval is based on results from the Chagas disease in children treated with nifurtimoxstudy, the first part of the largest Phase III program ever conducted in pediatric patients for the treatment of Chagas disease.

“In the study, Lampit showed good antiprotozoal activity in patients from 0 to 17 years old,” said Jaime Altcheh, MD, head of the Department of Parasitology and Chagas disease at the Ricardo Gutierrez Children’s Hospital in Buenos Aires, Argentina, and coordinating investigator of the Phase III trial.

The Phase III Lampit study was the first part of a prospective, randomized (to dosing regimen), double-blind evaluation of the efficacy, safety, and pharmacokinetics of nifurtimox in 330 children with Chagas disease.1 The study was conducted at 25 investigational sites in Argentina, Bolivia, and Colombia between 2016 and 2018.7 In the study, 330 pediatric patients with serologic evidence of T. cruzi infection (without Chagas disease-related cardiovascular and/or gastrointestinal symptoms) were randomly assigned in a 2:1 fashion to receive either a 60-day (n=219) or a 30-day (n=111) Lampit treatment regimen, and were followed up for one year after end of treatment.1 The results showed superiority in favor of the nifurtimox 60-day arm compared to the nifurtimox 30-day arm (not an approved dosing regimen). For additional clinical trial information, go to clinicaltrials.gov NCT02625974 and see full prescribing information. 

The study will continue with a second part (Lampit SECURE) to follow patients for an additional three years to confirm efficacy and safety.

Now that Lampit is approved for pediatric use in the U.S., Bayer is working to ensure access to the drug for all patients through retail channels. Commercially insured patients may qualify for a $0 co-pay to help with their out-of-pocket costs. For uninsured patients who cannot afford Lampit, the Bayer U.S. Patient Assistance Foundation, a charitable organization, will help eligible patients obtain the prescription medication at no cost. Restrictions apply. See Program Details for full information. Lampit is not approved in the U.S. for use in adults 18 years of age or older.

https://www.rxlist.com/lampit-drug.htm

FDA Approves Lampit (nifurtimox) for the Treatment of Chagas Disease in Children

FDA Approves Bavencio (avelumab) Plus Inlyta (axitinib) Combination for Patients with Advanced Renal Cell Carcinoma

In continuation of my update on axitinib

Merck KGaA, Darmstadt, Germany, which operates its biopharmaceutical business as EMD Serono in the US and Canada, and Pfizer Inc. (NYSE: PFE) today announced that the US Food and Drug Administration (FDA) has approved Bavencio (avelumab) in combination with Inlyta (axitinib) for the first-line treatment of patients with advanced renal cell carcinoma (RCC). This is the first FDA approval for an anti-PD-L1 therapy as part of a combination regimen for patients with advanced RCC. The approval of Bavencio in combination with Inlyta was based on positive results from the Phase III JAVELIN Renal 101 study (NCT02684006), in which the combination significantly improved median progression-free survival (PFS) compared with sunitinib by more than five months in the intent-to-treat (ITT) patient population (HR: 0.69 [95% CI: 0.56–0.84]; 2-sided p-value=0.0002; median PFS for Bavencio in combination with Inlyta: 13.8 months [95% CI: 11.1-NE]; sunitinib: 8.4 months [95% CI: 6.9-11.1]). The ITT population included patients regardless of PD-L1 expression and across IMDC (International Metastatic Renal Cell Carcinoma Database) prognostic risk groups (favorable 21%, intermediate 62% and poor 16%).

“As we look to continue to improve outcomes for people with advanced RCC, new treatment approaches have the potential to benefit patients,” said Robert J. Motzer, M.D., Jack and Dorothy Byrne Chair in Clinical Oncology, Memorial Sloan Kettering Cancer Center, New York, US, and principal investigator for JAVELIN Renal 101. “With today’s FDA approval of avelumab in combination with axitinib, we can now offer patients with advanced RCC a first-line treatment option that combines a PD-L1 immunotherapy with a well-known VEGFR TKI to provide a significant reduction in the risk of disease progression or death and doubling of the response rate compared with sunitinib.”

RCC is a type of cancer where PD-L1 expression may contribute to inhibition of the immune response against the tumor.2 It is also a highly vascular tumor, in which vascular endothelial growth factor (VEGF) plays a key role.3

“A kidney cancer diagnosis is life-changing for both patients and their loved ones, and having a treatment strategy for their disease quickly becomes a priority,” said Dena Battle, President, KCCure. “The approval of new treatments such as Bavencio in combination with Inlyta gives patients with advanced RCC much-needed options.”

There is a significant unmet need for first-line treatments that delay progression and have an acceptable safety profile. Approximately 20% to 30% of patients are first diagnosed with RCC at the advanced stage, and 30% of patients treated for an earlier stage go on to develop metastases.4,5 About half of patients living with advanced RCC do not go on to receive additional treatment after first-line therapy,6,7 for reasons that may include poor performance status or adverse events from their initial treatment.6,8,9

“Today’s approval of Bavencio in combination with Inlyta builds on Pfizer’s long heritage in bringing innovation to the RCC community with the hopes of making a significant and meaningful impact on the lives of patients,” said Andy Schmeltz, Global President, Pfizer Oncology. “For more than 12 years, Pfizer has led the field in its commitment to developing new treatments for patients with advanced kidney cancer.”

“With today’s FDA approval of Bavencio in combination with Inlyta, we feel privileged that we can offer patients with first-line advanced renal cell carcinoma a new treatment option,” said Rehan Verjee, President, EMD Serono, and Global Head of Innovative Medicine Franchises, Merck KGaA, Darmstadt, Germany.

In JAVELIN Renal 101, the objective response rate (ORR) was doubled in the ITT population with Bavencio in combination with Inlyta versus sunitinib (51.4% [95% CI: 46.6-56.1] vs. 25.7% [95% CI: 21.7-30.0]). With a median overall survival (OS) follow-up of 19 months, data for the trial’s other primary endpoint of OS were immature, with 27% of deaths in the ITT population, and the trial is continuing as planned. The most common adverse reactions (≥20%) were diarrhea, fatigue, hypertension, musculoskeletal pain, nausea, mucositis, palmar-plantar erythrodysesthesia, dysphonia, decreased appetite, hypothyroidism, rash, hepatotoxicity, cough, dyspnea, abdominal pain and headache. Serious adverse reactions occurred in 35% of patients receiving Bavencio in combination with Inlyta. The incidence of major adverse cardiovascular events (MACE) was higher with Bavencio in combination with Inlyta versus sunitinib.1 Findings from the study have been published in The New England Journal of Medicine.10

The European Medicines Agency (EMA) validated the Type II variation application for Bavencio in combination with Inlyta in advanced RCC in March 2019, and a supplemental application for Bavencio in combination with Inlyta in unresectable or metastatic RCC was submitted in Japan in January 2019.

The alliance is committed to providing patient access and reimbursement support through its CoverOne® program to patients who have been prescribed Bavencio. This program provides a spectrum of patient access and reimbursement support services intended to help US patients prescribed Bavencio receive appropriate access. CoverOne may be reached by phone at 844-8COVER1 (844-826-8371) or online at www.CoverOne.com.

Pfizer is committed to ensuring that patients who are prescribed Inlyta have access to this innovative therapy. Patients in the US have access to Pfizer Oncology Together™, which offers personalized support and financial assistance resources to help patients access their prescribed Pfizer Oncology medications. For more information, please call 1-877-744-5675 or visit PfizerOncologyTogether.com.

In an effort to streamline the patient enrollment process, EMD Serono and Pfizer have partnered to create a single enrollment form for the Bavencio and Inlyta combination for patients with advanced RCC that can be processed through both CoverOne and Pfizer Oncology Together. Each program will independently conduct the access and reimbursement activities for the product for which it is responsible.

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





FDA Approves Bavencio (avelumab) Plus Inlyta (axitinib) Combination for Patients with Advanced Renal Cell Carcinoma

FDA Approves Avaclyr (acyclovir ophthalmic ointment) for the Treatment of Herpetic Keratitis

  

 The US Food and Drug Administration (FDA) has approved Fera Pharmaceuticals' New Drug Application (NDA) for Avaclyr (acyclovir ophthalmic ointment) 3% for the treatment of herpetic keratitis. Orphan drug exclusivity was also granted, providing seven years of marketing exclusivity for the product.

“Avaclyr approval is a major milestone for the company,” noted Frank DellaFera, Fera’s founder and President. “Working closely with the eye care community we were able to identify a need for additional options to treat herpetic keratitis. Acyclovir is the gold standard treatment in herpes virus infections so it made sense to provide this therapy in an ophthalmic dosage form1. Fera is thankful to FDA for approving Avaclyr, making it available to physicians and patients in the US.”

Fera launch plans include finalizing its selection of a commercialization partner to provide physicians and patients access to this important therapy.

About Acute Herpetic Keratitis

According to the Center for Disease Control and Prevention (CDC), HSV (Herpes Simplex Virus) keratitis is an infection of the cornea—the clear dome that covers the colored part of the eye—that is caused by HSV. The infection usually heals without damaging the eye, but more severe infections can lead to scarring of the cornea or blindness. HSV keratitis is a major cause of blindness worldwide2-3.

About Avaclyr

Avaclyr (acyclovir ophthalmic ointment) 3%, is a herpes simplex virus nucleoside analog DNA polymerase inhibitor, is indicated in the treatment of acute herpetic keratitis (dendritic ulcers) in patients with herpes simplex (HSV-1 and HSV-2) virus. The most common adverse reactions (2-10%) reported in patients were eye pain (stinging), punctate keratitis and follicular conjunctivitis

https://www.drugs.com/newdrugs/fda-approves-avaclyr-acyclovir-ophthalmic-ointment-herpetic-keratitis-4939.html?utm_source=ddc&utm_medium=rss&utm_campaign=FDA+Approves+Avaclyr+(acyclovir+ophthalmic+ointment)+for+the+Treatment+of+Herpetic+Keratitis

FDA Approves Avaclyr (acyclovir ophthalmic ointment) for the Treatment of Herpetic Keratitis

FDA Approves Duaklir Pressair

In continuation of my update on formoterol 

Circassia Pharmaceuticals plc (“Circassia” or “the Company”; LSE: CIR), a specialty pharmaceutical company focused on respiratory disease,  announced the US Food and Drug Administration (FDA) has approved Duaklir for the maintenance treatment of chronic obstructive pulmonary disease (COPD). Duaklir is a fixed-dose combination of the long-acting muscarinic antagonist (LAMA) aclidinium bromide (400 mcg) and long-acting beta-agonist (LABA) formoterol fumarate (12 mcg) administered twice-daily via the breath-actuated inhaler Pressair®.  Circassia is on track to launch Duaklir in the United States in the second half of 2019 via its dedicated COPD sales force.

The Duaklir approval is based on a broad clinical database, including data from three phase III studies, ACLIFORM, AUGMENT and AMPLIFY. The label also includes clinical data from the phase IV ASCENT study, which shows aclidinium therapy is effective at reducing COPD exacerbations.  As a result, Duaklir® is the only twice-daily LAMA / LABA in the United States with COPD exacerbation data included in its prescribing information.

Steve Harris, Circassia’s Chief Executive, said: “We are delighted with the FDA approval of Duaklir, which we believe will provide a valuable treatment option for the significant number of patients with COPD in the United States. The addition of Duaklir to our portfolio further strengthens our range of marketed respiratory products and we look forward to launching it in the US in the coming months alongside our aclidinium monotherapy, Tudorza, as part of the significant LAMA / LABA market that is predicted to grow rapidly over the coming years.”

Michael Asmus, Circassia’s Vice President, US Medical Affairs, said: “With guidelines recommending combined LAMA and LABA therapy for a number of COPD patient groups, we believe Duaklir will make an important contribution to the treatment of this debilitating disease. Dukalir’s approval is based on a broad clinical database, including data demonstrating a reduction in the risk of COPD exacerbations driven by its aclidinium component, and we look forward to making this new therapeutic option available to patients across the United States.”

 Aclidinium bromide

  Formoterol

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

https://www.drugbank.ca/drugs/DB00983

FDA Approves Lonsurf (trifluridine/tipiracil) for Adult Patients with Previously Treated Advanced Gastric or Gastroesophageal Junction (GEJ) Adenocarcinoma

In continuation of my update on Lonsurf

Taiho Oncology, Inc.  announced that the United States Food and Drug Administration (FDA) has approved Lonsurf as a treatment for adult patients with metastatic gastric or gastroesophageal junction adenocarcinoma previously treated with at least two prior lines of chemotherapy that included a fluoropyrimidine, a platinum, either a taxane or irinotecan, and if appropriate, HER2/neu-targeted therapy.

“The approval of Lonsurf represents a significant milestone for patients living with advanced gastric or GEJ adenocarcinoma who have limited effective treatment options after standard treatment options have failed,” said Timothy Whitten, President and Chief Executive Officer, Taiho Oncology, Inc. “We thank all the patients and physicians who helped make this possible through their participation in Lonsurf clinical trials.”

The approval for Lonsurf follows an FDA Priority Review designation and is based on data from a global, randomized, Phase III TAGS trial evaluating Lonsurf plus best supportive care (BSC) versus placebo plus BSC in patients with previously treated advanced gastric cancer or GEJ adenocarcinoma following progression or intolerance to previous lines of standard therapy. The trial met its primary and secondary endpoints demonstrating prolonged overall survival (OS) with Lonsurf versus placebo, and a safety profile consistent with prior experience with this drug. Full results from the TAGS trial were presented at the European Society of Medical Oncology (ESMO) 2018 Congress with a simultaneous publication in The Lancet Oncology.1

“Effective treatments for patients with heavily pretreated advanced gastric and GEJ cancer are limited,” said Martin Birkhofer, MD, Senior Vice President and Chief Medical Officer, Taiho Oncology, Inc. “By improving survival, Lonsurf may provide a significant impact on the lives of these patients.”

This approval expands the current indication for Lonsurf in the United States, where it is currently approved for the treatment of patients with metastatic colorectal cancer (mCRC) who have been previously treated with standard chemotherapy, based on results obtained in the RECOURSE trial.

Ref : https://en.wikipedia.org/wiki/Trifluridine/tipiracil