FDA Grants Orphan Drug Status for ACE-083 to Treat Muscular Dystrophy

Treatment might be just around the corner for patients who suffer from facioscapulohumeral muscular dystrophy (FSHD). Recently, the US Food and Drug Administration has granted orphan drug designation to the Acceleron Pharmadrug, ACE-083. This drug might be able to treat an area of muscular dystrophy that currently has limited treatment options.

FSHD is a form of muscular dystrophy that mainly targets the muscles in the face, neck, arms, and trunk. Patients with this disease have difficulty with performing actions that involve their upper body, like whistling, using a straw, raising their arms, and doing sit-ups and pull-ups. They might even have difficulty closing their eyes and could also suffer from a curved spine. 

Once a patient begins to present these symptoms, doctors will ask a number of different questions and perform tests to rule out any other factors, like exposure to chemicals, surgeries, and injuries. Doctors will also use blood work, neurological tests, as well as muscle biopsies to confirm a diagnosis of FSHD. 

Currently, there is no known treatment for the disease, which means that patients with severe and advanced cases usually have a very uncomfortable lifestyle. As the disease progresses, patients can experience episodes of "malaise", or chronic burning pain, in their muscles, chronic fatigue, decreased lung function, and hearing loss. 

While FSHD is not fatal, it is a progressive genetic disease that usually targets patients in their early 20s. This disease can make their regular daily routine difficult to maintain. Because treatment options are so limited for FSHD, doctors prescribe patients to take anti-inflammatory drugs, or NSAIDs. Physical therapy and exercise also can help patients relieve their symptoms. 

This new designation of ACE-083 opens the door for multiple treatment options with FSHD. Read on to learn more about how this can help patients.