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Elevidys And The EMBARK Trial: Is Another Advisory Committee Meeting Coming?

 
• By Derrick Gingery

Sarepta says another public meeting is not necessary to expand the label for its DMD gene therapy, but the missed primary endpoint may require input from experts outside the FDA, and the situation renews the spotlight on CBER Director Peter Marks and his initial approval decision.

Sarepta
Sarepta will submit an sBLA requesting an expansion of the Elevidys label. • Source: Shutterstock

Another public discussion of Sarepta Therapeutics, Inc.’s gene therapy for Duchenne muscular dystrophy Elevidys may be on the horizon after the product missed the primary endpoint in its confirmatory study.

The company tried to spin the miss as a success, arguing that secondary endpoint data shows that Elevidys (delandistrogene moxeparvovec-rokl)...

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What UK’s Clinical Trials Overhaul Means For Orphan Drug Developers

 
• By Eliza Slawther

The UK government has acknowledged that sponsors of clinical trials for rare diseases face challenges around patient recruitment and trial design, which will be addressed in its clinical trials reform, a lawyer says.

Orphan Drug Trial Sponsors ‘Playing Catch-Up’ With Regulators – But Automation Can Help

 
• By Eliza Slawther

Clinical trial sponsors in the rare disease space face “big safety demands” from regulators, which can be challenging for those with limited resources. A CRO founder makes the case for “embedding automation” into the clinical trial process to keep pace with regulatory requirements.

Elegy For SACHRP: US HHS Research Protection Panel Tackled Tough Pediatric Trial Questions

 
• By Bridget Silverman

The termination of the HHS Secretary’s Committee on Human Research Protections closed an important forum for discussing pediatrics and pregnancy in clinical trial design, a former SACHRP chair said.

To Compete With China, US FDA Must Deregulate Early Gene Therapy Studies, CAR-T Inventor Says

 
• By Sarah Karlin-Smith

FDA regulation was painted as an obstacle to US dominance in the cell and gene therapy space even as panelists at an agency event praised the Office of Therapeutic Products' track record under Nicole Verdun.

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EUCOPE Wants Simpler Manufacturing Rules And Framework For Combined Trials Under EU Biotech Act

 
• By Eliza Slawther

EU authorities must “take advantage” of the upcoming Biotech Act to reassess the bloc’s policies and “offset existing challenges” for small and midsized enterprises, trade association EUCOPE has said.

Trial Cancellations And Delays Persist For CROs Amid Biotech Slowdown

 
• By Vibha Sharma

The whole contract research organization industry is being impacted by the current volatility in the pharmaceutical industry.

Citeline Japan Awards 2025 - Enter Now!

 
• By Ian Haydock and Lisa Takagi

The 14 July deadline for the Citeline Japan Awards 2025 in Tokyo is approaching fast, so here's a reminder to take a look at the categories and criteria and be sure to get your submissions in by this date. Sponsorship opportunities and tables for the event are also available.