1. Pink Sheet
  2. >>R&D
  3. >>Clinical Trials

Sarepta Confident Of Full US Approval For Duchenne Gene Therapy By Mid-2024

 
• By Andrew McConaghie

Elevidys has enjoyed a strong launch, despite being restricted to a small number of Duchenne patients. Sarepta is hoping the US regulator will overlook trial problems and allow its expanded use.

Sarepta building

Sarepta has reiterated its belief that its gene therapy Elevidys can gain a full US approval across all patients with Duchenne muscular dystrophy in 2024, despite falling short in a pivotal study last year.

Elevidys (delandistrogene moxeparvovec-rokl) won an accelerated US Food and Drug Administration approval in June to treat ambulatory patients ages four to five years old with a confirmed Duchenne gene mutation. This was based on Phase II studies, but its Phase III EMBARK confirmatory trial missed its primary endpoint in late October

Key Takeaways

  • Sarepta’s gene therapy racked up $200m in sales in six months, despite being restricted to a small group of Duchenne patients

  • Analysts had feared that the Phase III trial miss would mean the FDA would not grant full approval in a broader group of patients

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Clinical Trials

What The EMA Can Teach HTA Bodies About Joint Clinical Assessments

 
• By Francesca Bruce

EU-level joint clinical assessments conducted under the Health Technology Assessment Regulation need to be more flexible when it comes to evidence requirements, according to experts speaking at a gene and cell therapy conference.

EU ‘Can’t Expect Industry To Give Up Proprietary Insights’ Under Health Data & Clinical Trials Regulations

 
• By Eliza Slawther

Both the EU Clinical Trials Regulation and the European Health Data Space Regulation have the potential to improve harmonization and be highly valuable for industry – but the importance of protecting company data will be paramount, a life sciences consultant says.

UK Mandates Unmodified Standardized Contracts To Speed Clinical Trial Set-Up

 
• By Vibha Sharma

The policy expectation that the model clinical trial agreements are used without modification is in line requests from industry and the NHS/HSC.

DIA Korea: Medical Turmoil, Global Factors Blunt Korea’s Trial Edge

 
• By Jung Won Shin

DIA Korea hears how multiple domestic and global factors are affecting the environment for clinical trials in South Korea, and what actions may be taken to address this.

More from R&D

What The EMA Can Teach HTA Bodies About Joint Clinical Assessments

 
• By Francesca Bruce

EU-level joint clinical assessments conducted under the Health Technology Assessment Regulation need to be more flexible when it comes to evidence requirements, according to experts speaking at a gene and cell therapy conference.

EU ‘Can’t Expect Industry To Give Up Proprietary Insights’ Under Health Data & Clinical Trials Regulations

 
• By Eliza Slawther

Both the EU Clinical Trials Regulation and the European Health Data Space Regulation have the potential to improve harmonization and be highly valuable for industry – but the importance of protecting company data will be paramount, a life sciences consultant says.

How Tumult At US FDA Could Impact Merger and Acquisition Strategy

 
• By Joseph Haas and Nielsen Hobbs

With review and development uncertainly increasing, "it’s going to take longer for certain targets … to progress to a place where they’ve been derisked enough that big pharma is ready to write a big check," said Andrew Goodman of Paul Hastings.