Experimental Duchenne Muscular Dystrophy Treatment Earns Fast Track Designation

According to a story from PR Newswire, the biopharmaceutical company Avidity Biosciences, Inc., recently announced that its investigational therapy AOC 1044 has received the US Food and Drug Administration’s Fast Track designation. This treatment is being developed for people living with Duchenne muscular dystrophy with mutations that are responsive to exon 44 skipping. The company is focused on developing a class of RNA therapies called antibody oligonucleotide conjugates (AOCs).

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, boys are mostly affected, with girls 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 intended to accelerate the approval process for medicines that are intended to treat severe, life-threatening diseases and have the potential to fulfill a currently unmet medical need. This means that the drug must either treat a disease with no available treatment or show possible advantages over current treatments. Fast Track designation is requested by the developing company. Recipients are eligible for several benefits, such as more frequent interactions with the FDA to discuss the drug’s development, Rolling Review (in which the company can submit portions of the application by section instead of waiting for the entire application to be complete), more frequent written communication with the FDA, and potential eligibility for Accelerated Approval and Priority Review, if other criteria are met.

About the Trial

AOC 1044 is currently being evaluated in a Phase 1/2 trial and is the first of several AOC type therapies that the company hopes to develop for Duchenne muscular dystrophy. This trial is anticipated to include 24 patients and 40 healthy controls between ages 7-27. An optional open label extension study will be available to patients as well.

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