1. Pink Sheet
  2. >>Advanced Technologies
  3. >>Cell & Gene Therapies

Why Sarepta’s Gene Therapy Trial Enrollment Has Been More Successful Than Its Other DMD Therapies

 
• By Derrick Gingery

The EMBARK trial’s availability to more patients likely contributed to the much quicker enrollment than confirmatory trials for Exondys 51, Vyondys 53 and Amondys 45, which also saw enrollment targets and eligibility ages change over the years.

Duchenne muscular dystrophy
DMD advocates hope Sarepta's gene therapy candidate can push development in the disease forward. • Source: Shutterstock

The clinical trial testing Sarepta Therapeutics, Inc.’s gene therapy candidate in Duchenne muscular dystrophy enrolled remarkably faster than the company’s studies looking at three drugs that treat the rare disease, a likely function of its potential and a concern for the others’ futures.

SRP-9001 (delandistrogene moxeparvovec) is under priority review at the US Food and Drug Administration for accelerated approval. The EMBARK trial was first listed as recruiting on clinicaltrials

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 Cell & Gene Therapies

House Budget Bill Includes Delayed Orphan Fix, Also Risks Downstream Cell and Gene Coverage

 
• By Sarah Karlin-Smith

The first two classes of negotiated drugs under the Inflation Reduction Act would not benefit from a rare disease adjustment House Republicans included in their reconciliation package.

Keep Talking: US FDA’s Beleaguered Biologics Center Remains Vital To Regenerative Medicine

 
• By Bridget Silverman

Advisory committee for Capricor’s deriamocel planned, regenerative medicine advanced therapy designations proliferate for osteoarthritis, and the ranks of dual RMAT/breakthrough therapy designation holders grow.

Tougher Approval Standards May Follow Vinay Prasad’s Appointment To Lead US FDA’s CBER

 
• By Sarah Karlin-Smith

Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.

Sponsors Like START Rare Disease Pilot, Will Prasad Maintain Its Momentum?

 
• By Kate Rawson

Sponsors reported faster development times for products that joined the pilot program intended to speed rare disease treatments in CBER.

More from Advanced Technologies

EU Wants Industry To Define ‘Trade Secrets’ Under Health Data Sharing Regulation

 
• By Eliza Slawther

The European Health Data Space Regulation is deliberately “vague” when it comes to defining trade secrets because the EU wants the pharma industry to make recommendations on safeguarding intellectual property, a policy officer for the European Commission says.

Tougher Approval Standards May Follow Vinay Prasad’s Appointment To Lead US FDA’s CBER

 
• By Sarah Karlin-Smith

Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.

Sponsors Like START Rare Disease Pilot, Will Prasad Maintain Its Momentum?

 
• By Kate Rawson

Sponsors reported faster development times for products that joined the pilot program intended to speed rare disease treatments in CBER.