FDA Lifts Clinical Hold on Pfizer DMD Gene Therapy Linked to Patient Death

Pfizer said today it will open its first U.S. sites for a global Phase III trial assessing its fordadistrogene movaparvovec (PF-06939926) in Duchenne muscular dystrophy (DMD) after the FDA lifted a clinical hold on the company’s investigational new drug (IND) application for the program, which was imposed in December 2021 after a patient died in a Phase Ib open-label study of the mini-dystrophin gene therapy candidate.

Pfizer is among companies working to develop DMD gene therapies; others include Sarepta Therapeutics and Solid Biosciences.

On Wednesday, Solid said it concluded enrollment in its Phase I/II IGNITE DMD trial (NCT03368742) for SGT-001, and will continue monitoring dosed patients for five years post-treatment.

Here is the entire story: Genetic Engineering and Biotechnology News


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