Vamorolone

Satellos Reports Promising Efficacy Signals for Novel DMD Treatment in Adult Patients

3 hours ago

• Satellos Bioscience's investigational treatment for Duchenne muscular dystrophy (DMD) has demonstrated encouraging efficacy signals in adult patients, offering potential new options in a challenging therapeutic area. • The development comes amid setbacks in the DMD field, including Pfizer's withdrawal of its gene therapy fordadistrogene movaparvovec following a Phase III failure and a Phase II patient fatality. • The global DMD treatment market is projected to grow substantially from $2.3 billion in 2023 to $5.2 billion by 2033 across major markets, primarily driven by Elevidys and Santhera Pharmaceuticals' Agamree.

Wave Life Sciences to Seek FDA Approval for Duchenne Muscular Dystrophy Drug Following Promising Phase 2 Results

2 months ago

• 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.

FDA Approves Five Groundbreaking Treatments for Ultra-Rare Diseases in Recent Months

3 months ago

• The FDA has approved novel therapies for Duchenne Muscular Dystrophy, including ITF Therapeutics' Duvyzat (givinostat), which targets histone deacetylases to potentially slow muscle deterioration. • Breakthrough gene therapy Lenmeldy received approval for Metachromatic Leukodystrophy, offering the first targeted treatment option with significant survival benefits for pediatric patients. • Multiple rare diseases saw first-ever treatments, including Xolremdi for WHIM syndrome, dual approvals for Niemann-Pick Disease Type C, and Ctexli for Cerebrotendinous Xanthomatosis.

DMD Market Set to More Than Double to $5.2 Billion by 2033, Driven by Novel Therapies

3 months ago

• 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

4 months ago

• 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.

Catalyst Pharmaceuticals Expands Global Reach with FIRDAPSE Launch in Japan and AGAMREE Review in Canada

4 months ago

• DyDo Pharma has launched FIRDAPSE (amifampridine) in Japan for Lambert-Eaton myasthenic syndrome, marking Catalyst Pharmaceuticals' continued expansion of its rare disease portfolio globally. • Health Canada has accepted Kye Pharmaceuticals' New Drug Submission for AGAMREE with Priority Review status, potentially bringing the first treatment specifically indicated for Duchenne muscular dystrophy to Canadian patients by late 2025. • Catalyst Pharmaceuticals is strategically expanding its international footprint through sub-licensing partnerships while maintaining its commitment to improving access to novel therapies for rare disease patients worldwide.

Agamree Receives Positive Recommendation in Scotland and NICE Guidance in the UK for Duchenne Muscular Dystrophy

4 months ago

• 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.

Sarepta Seeks Accelerated Approval for DMD Gene Therapy SRP-9001

5 months ago

• Sarepta Therapeutics has submitted SRP-9001 (delandistrogene moxeparvovec) to the FDA for accelerated approval to treat ambulatory Duchenne muscular dystrophy (DMD) patients. • The filing is based on positive data from early-stage studies, showing improvements in clinical function and a consistent safety profile, while awaiting Phase 3 EMBARK results. • SRP-9001, a one-time gene therapy, delivers a shortened dystrophin gene via an AAV vector, addressing the underlying genetic defect in DMD patients. • If approved, SRP-9001 would offer a one-time treatment option for DMD, contrasting with Sarepta's existing chronic exon-skipping therapies.

Santhera's Agamree Receives NICE Recommendation and China NMPA Approval for Duchenne Muscular Dystrophy Treatment

5 months ago

• 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.

FDA Approves Santhera's Agamree (Vamorolone) for Duchenne Muscular Dystrophy

2 years ago

• 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.

Drug Development Updates