According to a story from Muscular Dystrophy News Today, a gene therapy in development by the pharma company Pfizer, Inc. was recently given Fast Track designation from the US Food and Drug Administration (FDA). This gene therapy, currently designated PF-06939926, is in development as a treatment for Duchenne muscular dystrophy, a rare genetic disorder. The therapy has also earned Orphan Drug status in the US and EU and the FDA’s Rare Pediatric Disease designation.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy is a neuromuscular disease, and it is one of the more severe types of muscular dystrophy. It is characterized by progressive muscle weakness that usually begins around age four and worsens quickly. As an X-linked genetic disease, males are mostly affected, with females only occasionally displaying mild symptoms. The disease is caused by mutations of the dystrophin gene. Symptoms of Duchenne muscular dystrophy include falling, abnormal walking posture, eventual loss of walking ability, muscle fiber deformities, intellectual disability (not in all cases), enlargement of the tongue and calf muscles, skeletal deformities, muscle atrophy, heart abnormalities, and difficulty with breathing. Treatment includes a variety of medications and therapies that can help alleviate symptoms and slow disease progression. Lifespan is usually into the thirties with good care. Better treatments for this disease are urgently needed. To learn more about Duchenne muscular dystrophy, click here.
About Fast Track Designation
Fast Track designation is generally reserved for therapies designed to address a medical condition or disease in which there is a major and urgent unmet need. The designation accelerates the pace of development. Therapies with this designation may also be eligible for other statuses, such as Accelerated Approval and Priority Review.
PF-06939926 is being developed as one-time gene therapy that is introduced directly into the bloodstream. This therapy uses an AAV viral vector to deliver a shortened but fully functional copy of the DMD gene to the muscles. Pfizer is hoping that this therapy could significantly slow or event halt the progression of muscle atrophy in Duchenne muscular dystrophy.
The granting of Fast Track status was based on encouraging data from a phase 1b study that is still ongoing. This study consists of 30 patients, and the treatment appears to be having beneficial effects.