Matt Winton’s Post

CALLING ALL RARE DISEASE ADVOCATES! LEND YOUR VOICE TO SUPPORT THE RARE DISEASE COMMUNITY IN TOMORROW'S MARKUP! The U.S. House of Representatives Energy and Commerce Committee will be holding a markup TOMORROW, June 27th, featuring TWO bills important to the rare disease community: the Give Kids a Chance Act (H.R. 3433) and the Telehealth Modernization Act (H.R. 7623). Now is the time to share your story and LEND YOUR VOICE.      Approximately 30 million Americans are living with rare diseases. The bills being voted on tomorrow will help address some of the common challenges rare disease patients face, including a lack of treatment options for children (who make up half of our community) and geographical barriers that prevent timely access to health care providers specializing in rare diseases.    Specifically, the Telehealth Modernization Act would extend Medicare's telehealth flexibilities granted during the pandemic for two more years. The Give Kids a Chance Act, which has been modified with helpful provisions to support the development of treatments to help kids living with rare diseases, including:  - Creating Hope Reauthorization Act (H.R. 7383) to reauthorize the Rare Pediatric Disease Priority Review Voucher program through September 30, 2029, a vital incentive supporting drug development for rare diseases that primarily affect children and often cut their lives far too short.  - Innovation for Pediatric Drugs Act (H.R. 6664) to update how research is currently funded as part of the Best Pharmaceuticals for Children Act (BPCA), which helps close data gaps around pediatric uses for approved drugs and strengthens FDA's ability to enforce post-market commitments around pediatric studies.  - RARE Act (H.R. 7384) to cement FDA's long-standing interpretation of how to properly award orphan drug exclusivity, a key drug development incentive established by the life-changing Orphan Drug Act. More than 95% of the 10,000 known rare diseases do not yet have any FDA-approved treatment and the 1 in 10 Americans living with rare diseases are counting on you to help amplify their voices. Together, we can make sure Congress hears our needs loudly and clearly, and our elected officials will know people are standing with them in their support of these bills.    HERE YOU CAN SEND A MESSAGE to your Representative TODAY on these two important priorities: https://lnkd.in/e73GvXfg

Good news for those of us with MAC! Mannkind, a company focused on the development and commercialization of innovative inhaled therapeutic products and devices for patients with endocrine and orphan lung diseases, announced… that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation of Clofazimine Inhalation Suspension (MNKD-101) for the treatment of nontuberculous mycobacterial (NTM) lung disease. Fast track designation is intended to facilitate the development, and expedite the review, of medicines to treat serious conditions and fill an unmet medical need. “We are pleased with the FDA’s decision to grant Fast Track designation for Clofazimine Inhalation Suspension, providing us an opportunity to accelerate our efforts to potentially bring an important medicine to patients living with NTM,” said Michael Castagna, PharmD, Chief Executive Officer of MannKind Corporation. “We are looking forward to the progression of the ICoN-1 study as well as an expedited review with a rolling submission.” ICoN-1 is a multi-national, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of Clofazimine Inhalation Suspension when added to guideline-based therapy in adults with refractory NTM lung disease caused by Mycobacterium Avium Complex (MAC), followed by an open-label extension. This single registrational study anticipates getting underway in June 2024 in the U.S., and internationally in the second half of 2024. The U.S. FDA previously designated Clofazimine Inhalation Suspension as both an orphan drug and a qualified infectious disease product (QIDP) for the treatment of pulmonary NTM infections. A drug that receives orphan drug exclusivity receives seven years of exclusivity, and one that also earns QIDP designation, may receive an additional five years of market exclusivity. Source: Morningstar #MACLungDisease #NTMLungDisease #NTM #Research #GoodNews #EvidencebasedResearch MannKind Corporation

New Request for Applications for FDA Rare Neurodegenerative Disease Grant Program Deadline to submit applications: October 22, 2024 The U.S. Food and Drug Administration (FDA) announced a new funding opportunity for the FDA Rare Neurodegenerative Disease Grant Program to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare neurodegenerative diseases for children and adults. Through the support of collaborative, efficient, and innovative clinical trials, FDA hopes to exert a broad and positive impact by increasing the number of approved medical products for rare neurodegenerative diseases. The FDA Rare Neurodegenerative Disease Grant Program was established by the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act. This program is administered by FDA’s Office of Orphan Products Development to promote medical product development for rare neurodegenerative diseases such as ALS.  #fda #rarediseases #program #neurodegenerative #acceleratedaccess #deadline https://lnkd.in/g_SQN-2s

NEWS 📰 First patient dosed in Assembly Biosciences’ phase 1b trial for hepatitis B treatment – [Pharmafile] 🔊Anuj Gaggar MD PhD, chief medical officer of Assembly Biosciences, commented: “We believe ABI-4334 has the potential to demonstrate best-in-class antiviral activity in line with the very high potency seen preclinically and are pleased to initiate dosing for this differentiated investigational therapy in individuals with chronic HBV. In this 28-day placebo-controlled study, we will be able to measure the impact of escalating doses of ABI-4334 on HBV DNA and refine the projected activity against cccDNA formation, along with generating key safety data. We look forward to reporting interim results from the study later this year.” 📲 https://ow.ly/J27c50SmKFh

FDA approves monoclonal antibody for children with eosinophilic esophagitis!   Congratulations to Regeneron and Sanofi for the approval of Dupixent (dupilumab), the first and only treatment for children aged 1 year and older with eosinophilic esophagitis (EoE). Dupixent is a fully human monoclonal antibody and works by inhibiting IL-4 and IL-13 pathway signaling.   EoE is a chronic, progressive disease driven where inflammation damages the esophagus and impairs its function. Currently, about 21,000 children under the age of 12 in the U.S. are being treated for EoE with unapproved therapies.   Read the press release here: https://lnkd.in/ejQtiqB2   #Biointron #Antibodies #Immunotherapy #PharmaNews #DrugDevelopment #FDA #Monoclonal #Research #Therapeutics

Johnson & Johnson Innovative Medicine announced that, in the Phase 3 VIVACITY study in generalized myasthenia gravis (gMG), nipocalimab met the primary endpoint, achieving statistically significant reduction in MG-ADLa score from baseline over weeks 22 to 24 compared with placebo. Nipocalimab is an investigational, high-affinity, fully human, aglycosylated, effectorless, anti-FcRn monoclonal antibody. gMG is a chronic, life-long, rare, and highly debilitating autoantibody-driven neuromuscular disease characterized by fluctuating muscle weakness. As next steps, Johnson & Johnson plans to present full results from the Phase 3 VIVACITY study at an upcoming scientific medical congress and engage with global regulatory authorities about bringing nipocalimab to patients living with gMG. #mabs https://lnkd.in/efyrPBhp

#FDAapproved X4 Pharmaceuticals’ #newdrug #mavorixafor #Xolremdi, on 4/29/2024, to increase the number of circulating mature neutrophils and lymphocytes in patients 12 and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis). Mavorixafor inhibits CXCR4 leading to an increase in circulating neutrophils and lymphocytes. The most common adverse reactions with mavorixafor are thrombocytopenia, pityriasis, rash, rhinitis, epistaxis, vomiting, and dizziness. Approval of mavorixafor was based on the 52-week, 31 patient, Phase III, 4WHIM trial, where patients treated with mavorixafor achieved a time above Threshold-Absolute Neutrophil Count (TAT-ANC) of at least 500 Cells/Microliter of 15 hours compared to 2.8 hours with placebo in patients with WHIM syndrome. The increase in neutrophils lead to a reduction in infection frequency, severity, duration, and antibiotic use. (Blood. 2024 Apr 21:blood.2023022658. doi: 10.1182/blood.2023022658. Epub ahead of print) Annual WAC for mavorixafor was set at $496,400 for patients weighing over 50 kg and $372,300 for 50 kg or less. #prescriberight #pharmacists #physicians #nurses

In the UK alone, 3.5 million people are living with a rare disease – and 1 in 17 people will be affected by a rare disease at some point in their lifetime.   That is why development of effective therapies in the rare/orphan disease sector is so important. While the diseases might be rare, there are millions of people that require access to novel and potentially life-saving treatments.   Check out the recent article in the Open Access Government where Gillian Molloy, Director of Market Access, AscellaHealth provides insights into the specialty pharmaceutical and rare disease industry.   Read the full article here:   https://lnkd.in/eQfQe7Ve   #RareDiseases #DrugTreatment  

On August 14, the U.S. Food and Drug Administration (FDA) approved #axatilimab-csfr (Niktimvo), a colony-stimulating factor–1 receptor–blocking antibody, for the treatment of chronic graft-vs-host disease (#GVHD) after failure of at least #two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. #AGAVE-201 Efficacy was evaluated in AGAVE-201 (ClinicalTrials.gov identifier #NCT04710576), a randomized, open-label, multicenter trial investigating three dosages of axatilimab in adult and pediatric patients with recurrent or refractory chronic GVHD who had received at least #two lines of systemic therapy and required additional treatment. The major efficacy outcome measure was overall response rate through cycle 7 day 1, where overall response included complete or partial response according to the 2014 National Institutes of Health Consensus Development Project on Response Criteria. The overall response rate was 75% The median time to first response was 1.5 months The median duration of response, calculated from first response to disease progression, death, or new systemic therapies for chronic GVHD, was 1.9 m

The Phase II Clinical Study of Grand Pharma’s Global Innovative Product STC3141 Has Completed the First Patient Enrollment and Dosing in China The Phase II clinical study for the treatment of sepsis in China of the global innovative drug STC3141, which is developed by the Grand Pharma Group Limited (0512.HK)’s wholly-owned subsidiary Grand Medical Pty Ltd. (an innovative drug R&D center set up by the Group in Australia), has recently completed first patient’s enrollment and dosing recently. This is another significant R&D progress for the Group in the field of respiratory and severe disease anti-infection. The clinical study is a multi-center, randomized, double-blinded, placebo-controlled Phase II dose-exploring clinical study, aiming to evaluate the efficacy, safety and pharmacokinetic characteristics of STC3141 at different doses in patients with sepsis. The success of STC3141 in multiple clinical studies in the treatment of sepsis, ARDS and severe COVID-19 infection has revealed the favorable safety, tolerability and clinical benefit potential of this product in the treatment of severe diseases. STC3141 and APAD, another global innovative product for sepsis of the Group, complement each other in mechanism of action and are expected to form a good product portfolio in the treatment of severe diseases such as sepsis.