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Pfizer DMD Gene Therapy Phase III Trial Faces A US Delay; Can Sarepta Close The Gap?

Sarepta Hopes To Meet With FDA Midyear

 
• By Jessica Merrill

Pfizer disclosed that a Phase III trial for the gene therapy has been unable to start in the US because of questions from the FDA, even while enrollment in 15 sites outside the US is under way.

Boat Race
Pfizer and Sarepta are in a race to develop a DMD gene therapy • Source: Alamy

Pfizer Inc., which has taken the lead in the race to bring the first gene therapy to market for Duchenne muscular dystrophy (DMD), has faced a delay with the start of a Phase III trial in the US, presenting an opportunity for Sarepta Therapeutics, Inc. to close the gap with its rival gene therapy, SRP-9001.

Pfizer has been the first to advance its gene therapy candidate – fordadistrogene movaparvovec – into Phase III clinical development, but US enrollment has been held up by the US Food and Drug Administration, the company disclosed during its first quarter sales and

More from Gene Therapies

Which Firms Are Most Exposed To Prasad’s Possible Accelerated Approvals Clampdown?

 
• By Andrew McConaghie

Vaccine and accelerated approvals are expected to face greater scrutiny under new CBER leader Vinay Prasad, but he has promised “no light switch change” to policy, giving some reassurance to companies whose share prices have been battered.

Abeona Sets $3.1m Price For Rare Skin Disorder Gene Therapy

 
• By Joseph Haas

Abeona plans to offer an outcomes-based payment model for Zevaskyn, which likely will see complementary use with Krystal’s Vyjuvek in recessive dystrophic epidermolysis bullosa patients.

Verve Moves Toward Lilly Opt-In For PCSK9 Program With Phase Ib Results

 
• By Alaric DeArment

The company posted positive initial results from the Heart-2 trial in HeFH and CAD, with strong LDL cholesterol lowering and a clean safety profile.

Ferring’s Faith In Bladder Cancer Gene Therapy Starts To Pay Off

 
• By Kevin Grogan

Adstiladrin sales hit €70m in first full year on the market

More from Advanced Therapies

Gilead Pipeline Progress Offsets Oncology Sales Slowdown

 
• By Alaric DeArment

Sales of the company’s approved cancer therapies were weaker or down, but Trodelvy is likely to see a label expansion and cell therapies are making progress.

In Brief: BriaCell’s Breast Cancer Cell Therapy Boosts Survival In Phase II

 
• By Sushmita Panda

BriaCell announced its cell therapy Bria-IMT plus checkpoint inhibitors bested Gilead’s Trodelvy in a Phase II trial in HR+ breast cancer; a Phase III trial is enrolling.

Verve Moves Toward Lilly Opt-In For PCSK9 Program With Phase Ib Results

 
• By Alaric DeArment

The company posted positive initial results from the Heart-2 trial in HeFH and CAD, with strong LDL cholesterol lowering and a clean safety profile.