Experimental Treatment for Charcot-Marie-Tooth Disease Gets FDA Fast Track Designation

According to a story from Digital Journal, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that its investigational drug candidate ACE-083 was granted Fast Track designation from the US Food and Drug Administration. ACE-083 is in development for the treatment of the rare neuromuscular disorder Charcot-Marie-Tooth disease (CMT). Acceleron is dedicated to the development of TGF-beta therapies to treat serious and rare diseases.

About Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth disease is a hereditary disorder of the peripheral nervous system. It is most characterized by a progressive loss of touch sensation and muscle tissue in several different parts of the body. The cause of this disease is usually linked to a duplication of a certain region of chromosome 17 that affects the PMP22 gene. There are multiple types of Charcot-Marie-Tooth disease, with all types aside from type 2 having a demyelinization effect. Type 2 causes damage to the neuronal axon instead. Symptoms include foot drop, muscle wasting (typically in the arms, legs, and hands), painful muscle spasms, loss of sensation in the limbs, scoliosis, trouble speaking, chewing, swallowing, and tremors. Treatment typically includes therapy and surgery in order to maintain function. There is no cure. The disease can occur early in life or as late as the 30s and 40s. To learn more about Charcot-Marie-Tooth disease, click here.

About Fast Track Designation

Fast Track designation is a mechanism implemented by the FDA which helps facilitate the efficient development of therapies that treat serious diseases and fulfill a medical need that is not being met by current therapies. A drug can also receive Fast Track if it displays substantial advantages in safety or effectiveness over currently available treatments. A company that receives the designation will more closely collaborate with the FDA in both in-person meetings and written correspondence to aid in the drug’s development.

The drug is also eligible for rolling review, which allows the company to submit portions of the New Drug Application (NDA) as they are completed instead of waiting until the entire application is finished. If relevant criteria are met, the therapy may also be eligible for Accelerated Approval and Priority Review.

The designation is the result of favorable interim data in ongoing Phase 2 trials of ACE-083 in which the drug is being tested against Charcot-Marie-Tooth disease and facioscapulohumeral muscular dystrophy (FSHD). Results show that the drug has the potential to increase muscle volume.


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