Pfizer Inc. announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne has received Fast Track designation from the FDA.

PF-06939926 is currently being evaluated to determine the safety and efficacy of this gene therapy in people with Duchenne.

The Fast Track designation is a process designed to facilitate the development and expedited review of drugs that treat serious conditions and fill unmet medical needs.

Read the full update from Pfizer.

Read Pfizer’s update:

Pfizer Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Investigational Gene Therapy

Thursday, October 01, 2020 – 06:45am

NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE: PFE) today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the U.S. Food and Drug Administration (FDA). PF-06939926 is currently being evaluated to determine the safety and efficacy of this gene therapy in boys with DMD.

Fast Track is a process designed to facilitate the development, and expedite the review, of new drugs that are intended to treat or prevent serious conditions that have the potential to address an unmet medical need. This designation was granted based on data from the Phase 1b study that indicated that the intravenous administration of PF-06939926 was well-tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

“The FDA’s decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy,” said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. “DMD is a devasting condition and patients, and their parents, are waiting desperately for treatment options. We are working to advance our planned Phase 3 program as quickly as possible.”

DMD is a devastating and life-threatening X-linked disease that is caused by mutations in the gene encoding dystrophin, which is needed for proper muscle membrane stability and function. Patients present with muscle degeneration that progressively worsens with age to the extent that they require wheelchair assistance when they are in their early teens, and unfortunately, usually succumb to their disease by the time they are in their late twenties. It is estimated that there are ~10-12,000 individuals affected with DMD in the US.

About PF-06939926

PF-06939926 is an investigational, recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The rAAV9 capsid was chosen as the delivery vector because of its potential to target muscle tissue. Pfizer initiated the Phase 1b multi-center, open-label, non-randomized, ascending dose study of a single intravenous infusion of PF-06939926 in 2018. The goal of the study is to assess the safety and tolerability of this investigational gene therapy. Other objectives of the clinical study include measurement of dystrophin expression and distribution, as well as assessments of muscle strength, quality and function.