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Can Sarepta Beat Pfizer To A Duchenne Gene Therapy Breakthrough?

New Updates Sway Sentiment Behind Sarepta

 
• By Andrew McConaghie

As rival Phase III gene therapy trials get underway, Sarepta’s product could have the edge in safety and efficacy over its rival from Pfizer, but the chance of failure remains high.

Sarepta Building 2
Sarepta's is already earning revenues from its three exon-skipping DMD drugs, but a gene therapy could represent a true breakthrough for patients and their families. • Source: Alamy

Sarepta has become a company that other biopharma companies love to hate, thanks to it having received US Food and Drug Administration approval for three Duchenne muscular dystrophy (DMD) drugs, despite not having (yet) generated Phase III efficacy data for them. (Also see "Sarepta’s Next-Generation Drugs Draw Attention Amid ‘Incremental’ Approval" - Scrip, 26 February, 2021.)

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