FDA Lifts Clinical Hold on Pfizer DMD Phase 3 Clinical Trial

The FDA has lifted a clinical hold on Pfizer’s phase 3 clinical trial of its investigational gene therapy, fordadistrogene movaparvovec, in patients with Duchenne muscular dystrophy (DMD).

The agency halted the study in December after a patient with nonambulatory DMD died in a phase 1b study of the recombinant adeno-associated virus serotype 9 (AAV9) capsid gene therapy, which delivers a shortened version of the human dystrophy gene.

Pfizer will go ahead with the trial for ambulatory DMD patients using a new protocol that includes a seven-day hospitalization period to enable close monitoring and management of patients following administration of the gene therapy.

The company said it will be looking into new treatment options for nonambulatory DMD patients, who will no longer be part of the drug’s phase 3 study.