Pfizer Inc’s investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the US Food and Drug Administration (FDA), it announces.

The gene therapy is currently being evaluated to determine its safety and efficacy 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, a media release from Pfizer Inc notes.

“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.

“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.”

— Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development

About the Drug

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.

[Source(s): Pfizer Inc, Business Wire]


Related Content:
FDA Approves Targeted Treatment for Duchenne Mutation
Nationwide Children’s Hospital Begins Human Trial of Gene Therapy for Duchenne Mutation
Gene Therapy Trial in Duchenne Kids Suggests Functional Improvement Gains