Investigational Drug for Charcot-Marie-Tooth Disease Earns Orphan Drug Designation

According to a story from Central Charts, the biopharmaceutical company Acceleron Pharma Inc. recently announced that the company’s experimental drug candidate ACE-083 has earned Orphan Drug designation from the US Food and Drug Administration (FDA). The drug is in development as a treatment for Charcot-Marie-Tooth disease. Acceleron is focused on the development of therapies for 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 genetic mutation, but the mutation involved varies depending on the variant of Charcot-Marie-Tooth disease. 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 Orphan Drug Designation

Orphan Drug designation is an incentive program that is reserved for therapies and drugs that are in development as treatments for diseases that are considered rare. This is defined as any illness that affects less than 200,000 people in the US. To qualify for the designation, a drug must demonstrate superiority in safety or effectiveness over currently available treatments or it must satisfy a currently unmet medical need. The designation confers several benefits for the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the drug is approved.

About ACE-083

The earning of this designation is a significant development milestone for ACE-083, which is a locally acting muscle agent that utilizes Myostatin+ in order to maximize muscle strength and growth in the areas where the therapy is administered. ACE-083 may have clinical relevance in other diseases that cause muscle atrophy beyond Charcot-Marie-Tooth disease.


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