Wave Life Sciences to Seek FDA Approval for Duchenne Muscular Dystrophy Drug Following Promising Phase 2 Results
• Wave Life Sciences will pursue FDA accelerated approval for WVE-N531, an exon 53 skipping therapy for Duchenne muscular dystrophy, following positive Phase 2 trial results showing increased dystrophin production.
• The experimental treatment demonstrated substantial improvements in muscle health with a statistically significant improvement in "time to rise" functional tests compared to historical controls through 48 weeks of treatment.
• If approved, WVE-N531 could offer a potential monthly dosing regimen and become a new therapeutic option for approximately 8-10% of Duchenne patients with specific genetic mutations amenable to exon 53 skipping.
DMD Market Set to More Than Double to $5.2 Billion by 2033, Driven by Novel Therapies
• The Duchenne muscular dystrophy market across seven major markets is projected to grow from $2.3 billion in 2023 to $5.2 billion by 2033, fueled by recent approvals of Elevidys and Agamree.
• Exon-skipping therapies currently dominate the market with $1.0 billion in sales, expected to reach $1.8 billion by 2033, while gene therapies are forecast to contribute $821 million.
• The United States maintains market leadership with 84.8% share, though significant unmet needs persist for non-ambulatory patients and those requiring treatment for late-stage complications.
Entrada Therapeutics Advances DMD Treatment: UK MHRA Approves Phase I/II Trial for Novel Exon-Skipping Therapy
• Entrada Therapeutics has received MHRA authorization to commence ELEVATE-44-201, a Phase I/II trial evaluating ENTR-601-44 for Duchenne muscular dystrophy patients with exon 44 skipping mutations.
• The global trial consists of two parts: a multiple ascending dose study with 24 subjects receiving 6-18mg/kg doses, followed by an efficacy and safety assessment phase with quality-of-life measures.
• ENTR-601-44, an Endosomal Escape Vehicle-conjugated therapy, aims to restore functional dystrophin protein production, with trial initiation planned for Q2 2024.
Agamree Receives Positive Recommendation in Scotland and NICE Guidance in the UK for Duchenne Muscular Dystrophy
• The Scottish Medicines Consortium (SMC) has accepted Agamree (vamorolone) for use within NHS Scotland for treating Duchenne muscular dystrophy (DMD) in patients aged four and older.
• NICE has issued positive final guidance recommending Agamree for use in the NHS in England, Wales, and Northern Ireland for DMD patients aged four and older.
• Agamree is the first medicinal product for DMD to receive full approval in the EU, US, and UK, offering clinically important tolerability benefits compared to standard corticosteroids.
• The approvals and recommendations are based on studies like VISION-DMD, which demonstrated Agamree's efficacy and safety profile, marking a significant advancement in DMD treatment.
Santhera's Agamree Receives NICE Recommendation and China NMPA Approval for Duchenne Muscular Dystrophy Treatment
• The National Institute for Health and Care Excellence (NICE) has recommended Agamree (vamorolone) for treating Duchenne muscular dystrophy (DMD) in patients aged four and older in England, Wales, and Northern Ireland.
• China's National Medical Products Administration (NMPA) has approved Agamree for DMD, marking it as the first approved treatment for the condition in China.
• Agamree demonstrated clinically significant safety benefits, including better bone metabolism and growth, compared to standard corticosteroids in clinical trials.
• Santhera is collaborating with NHS and Sperogenix Therapeutics to ensure rapid patient access to Agamree in the UK and China, respectively.
NCFB Pipeline Shows Promise with Over 15 Companies Developing New Therapies
• The non-cystic fibrosis bronchiectasis (NCFB) market is experiencing growth due to rising prevalence and increased awareness, leading to early diagnosis and treatment.
• Over 15 companies are actively developing more than 15 NCFB drugs, aiming to improve the treatment landscape for this chronic lung condition.
• Key players like Insmed, AstraZeneca, and Verona Pharma are advancing promising therapies such as brensocatib, benralizumab and ensifentrine through clinical trials.
• Recent clinical trial milestones, including Phase III results for brensocatib and Phase II enrollment for AP-PA02, signal progress in addressing unmet needs in NCFB treatment.
DMD Pipeline Shows Promise with Over 75 Therapies in Development
• Over 75 drugs are in development for Duchenne Muscular Dystrophy (DMD), targeting various mechanisms and routes of administration.
• REGENXBIO initiated a Phase I/II trial of RGX-202 in young boys with DMD to assess safety and efficacy.
• Emerging therapies focus on gene modulation, dystrophin replacement, and anti-inflammatory approaches to combat DMD.
• Key companies like Sarepta Therapeutics, Italfarmaco, and FibroGen are advancing DMD therapeutics through clinical trials.
Vamorolone Demonstrates Dual Mechanism as Mineralocorticoid Receptor Antagonist in LIONHEART Study
• Vamorolone (Agamree) showed a statistically significant increase in the urinary sodium/potassium ratio compared to placebo in the LIONHEART study, indicating mineralocorticoid receptor antagonism.
• The study reveals vamorolone's dual mechanism as both a mineralocorticoid receptor antagonist and a glucocorticoid receptor agonist, distinguishing it from traditional corticosteroids.
• These findings suggest potential cardioprotective benefits of vamorolone for Duchenne muscular dystrophy (DMD) patients, warranting further investigation into long-term cardiac outcomes.
• Vamorolone is already FDA-approved for DMD, and ongoing research aims to confirm its benefits in managing cardiac complications, especially when initiated early.
Swissmedic Accepts Santhera's Marketing Application for AGAMREE® in Duchenne Muscular Dystrophy
• Santhera Pharmaceuticals announced that Swissmedic has accepted the marketing authorization application for AGAMREE® (vamorolone) for treating Duchenne muscular dystrophy (DMD).
• Swissmedic will assess AGAMREE® under Article 13 TPA, considering the EU approval from December 2023 to streamline the approval process.
• AGAMREE® has already been approved in the U.S., EU, and UK, and is under priority review in China, addressing the unmet need for improved DMD treatments.
• The outcome of the Swissmedic review is expected in late H1-2026, with a possibility of acceleration to early 2026 if Article 13 is accepted.
FDA Approves Santhera's Agamree (Vamorolone) for Duchenne Muscular Dystrophy
• The FDA has approved Agamree (vamorolone) for treating Duchenne Muscular Dystrophy (DMD) in patients aged two years and older, offering a novel corticosteroid treatment option.
• Agamree demonstrated comparable efficacy to standard corticosteroids in clinical trials, with data suggesting a reduction in adverse events related to bone health, growth, and behavior.
• Catalyst Pharmaceuticals holds the exclusive North American license to commercialize Agamree and plans to launch the product in Q1 2024 with a patient assistance program.
• The approval is based on data from the Phase 2b VISION-DMD study and supported by safety information from open-label extension studies, highlighting its potential to improve the quality of life for DMD patients.
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