Vesleteplirsen

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.

Sarepta Therapeutics Halts Development of Vesleteplirsen for Duchenne Muscular Dystrophy

6 months ago

• Sarepta Therapeutics has discontinued the development of vesleteplirsen, an experimental exon 51-skipping therapy. • The decision impacts Duchenne muscular dystrophy patients with specific mutations who were potential beneficiaries. • This strategic shift alters Sarepta's approach to next-generation treatments for this genetic muscle-wasting disease.

Sarepta Halts Development of Vesleteplirsen for Duchenne Muscular Dystrophy

6 months ago

• Sarepta Therapeutics discontinues the development of vesleteplirsen (SRP-5051), an exon-skipping antisense oligonucleotide (ASO) for Duchenne muscular dystrophy (DMD). • The decision was influenced by an updated risk-benefit analysis, FDA feedback, and the evolving therapeutic landscape for DMD. • Vesleteplirsen, designed as a more potent version of eteplirsen, showed increased dystrophin expression but faced safety concerns, including low magnesium levels and decreased kidney function. • Sarepta will focus on other molecular candidates, including late-stage gene therapies for limb-girdle muscular dystrophy.

Sarepta Discontinues Development of Vesleteplirsen for Duchenne Muscular Dystrophy

6 months ago

• Sarepta Therapeutics halts development of vesleteplirsen (SRP-5051), a more potent follow-up to Exondys 51 for Duchenne muscular dystrophy (DMD). • The decision was influenced by risk-benefit considerations, FDA feedback, and the evolving DMD treatment landscape, including gene therapy options. • Vesleteplirsen faced challenges, including a clinical hold by the FDA due to hypomagnesaemia observed in a clinical trial. • Sarepta's revenues are shifting towards Elevidys, its gene therapy for DMD, which saw $181 million in sales compared to $249 million for exon-skipping drugs.

Sarepta Therapeutics Halts Development of Vesleteplirsen for Duchenne Muscular Dystrophy

7 months ago

• Sarepta Therapeutics discontinues vesleteplirsen (SRP-5051) development for Duchenne muscular dystrophy due to safety concerns and FDA feedback. • The decision follows a Phase 2 trial hold in 2022 due to hypomagnesemia and kidney function decrease observed in some patients. • Vesleteplirsen, an enhanced version of eteplirsen, aimed to improve dystrophin production via exon 51 skipping but faced tolerability issues. • Sarepta will focus on other RNA-based therapies and gene therapies for Duchenne and limb-girdle muscular dystrophy.

Sarepta Discontinues Next-Gen DMD Drug Vesleteplirsen Amidst Regulatory Hurdles and Elevidys Success

7 months ago

• Sarepta Therapeutics halts the development of vesleteplirsen, a next-generation exon 51-skipping therapy for Duchenne muscular dystrophy, after FDA feedback and internal analysis. • The decision follows concerns about persistent hypomagnesemia and the FDA's indication that the accelerated approval pathway is not viable for vesleteplirsen. • Elevidys, Sarepta's gene therapy for DMD, demonstrates strong commercial performance with $181 million in net revenue in Q3, exceeding expectations. • Sarepta's total revenue for Q3 reaches $467.2 million, marking a significant increase from $331.8 million in the same period of the previous year.

Sarepta Therapeutics Reports Strong Q3 2024 Financials and ELEVIDYS Expansion

7 months ago

• Sarepta Therapeutics reported a significant increase in total revenues for Q3 2024, reaching $1,243.6 million, up from $846.6 million in the same period last year. • ELEVIDYS (delandistrogene moxeparvovec-rokl) received FDA approval for treating ambulatory Duchenne muscular dystrophy patients and expanded use to non-ambulatory patients. • The company is advancing its manufacturing capabilities and expanding its product pipeline through strategic initiatives, including a new R&D facility and partnerships. • Sarepta faces challenges including regulatory compliance, reimbursement uncertainties, and market acceptance of gene therapy, impacting its operational performance.

Duchenne Muscular Dystrophy: Biotech Firms Advance Novel Therapies

8 months ago

• Wave Life Sciences reported positive interim results for WVE-N531, an exon-skipping oligonucleotide, showing promising dystrophin expression in patients with Duchenne muscular dystrophy. • Sarepta Therapeutics expanded the label for its gene therapy Elevidys to include all patients aged four and older with mutations in the dystrophin gene. • Italfarmaco's Duvyzat (givinostat), the first nonsteroidal treatment for all genetic variants of Duchenne, received FDA approval based on phase 3 trial results. • Avidity Biosciences' delpacibart zotadirsen (del-zota) demonstrated a significant increase in dystrophin production and reduction of creatine kinase levels in a phase 1/2 trial.

Dyne Therapeutics' DYNE-251 Shows Promising Dystrophin Expression and Functional Improvements in Duchenne Muscular Dystrophy Trial

9 months ago

• DYNE-251 demonstrated a mean absolute dystrophin expression of 3.71% of normal, over 10-fold higher than eteplirsen, in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. • Clinical data from the DELIVER trial indicated meaningful improvements in motor function, including the Stride Velocity 95th Centile, meeting clinically important benchmarks defined by the European Medicines Agency. • The investigational therapy exhibited a favorable safety profile, with most treatment-emergent adverse events being mild or moderate, supporting its potential for long-term use. • Dyne Therapeutics is initiating registrational cohorts in the DELIVER trial and plans to provide an update on the path to registration for DYNE-251 by the end of 2024.

Drug Development Updates