Acceleron Pharma has just received Fast Track designation from the US Food and Drug Administration (FDA) for their experimental treatment ACE-083 designed to treat patients with facioscapulohumeral muscular dystrophy (FSHD). You can read the full press release here, at BioPortfolio.

FSHD is a genetic condition that causes muscle weakness that predominantly affects the upper body. It is a rare disease, and it’s estimated that approximately 20,000 people in the US have it. The onset of symptoms usually occurs before age 20 and tends to affect muscles in a descending pattern, beginning at the head and upper body and gradually progressing downwards. Some medical professionals divide FSHD into two forms: adult onset and infantile onset. Both types usually first appear before 20, but infantile onset occurs in younger children, is generally more severe, and can be associated with hearing and vision loss. FSHD progresses slowly, and most people with the condition will have a normal lifespan. Treatment options for those affected are limited, but researchers are working on developing better medicines.

The experimental new treatment, ACE-083, is hoped to help patients with FSHD by improving muscle strength and function in specific muscles targeted by the drug. It works by inhibiting specific proteins that reduce muscle growth. The targeted proteins are part of a group called TGF-beta proteins and include molecules such as myostatin and activins. Inhibiting these proteins should cause more muscle growth and strength in the areas where the drug is administered. This approach, which only targets particular muscles, should limit the side effects of the treatment.

ACE-083 has already undergone a Phase 1 trial, which produced encouraging results. It is currently being further tested in a Phase 2 trial in patients with FSHD, and researchers have announced that the study seems to be going well. Based on these results, the FDA awarded ACE-083 Fast Track designation. This is reserved for drugs that seem to have the potential to meet a significant unmet patient need, and it is designed to speed up their development through close communication between the pharmaceutical company and the FDA in order to bring the treatment to patients more quickly.

In addition to its use for FSHD, the experimental drug ACE-083 is also undergoing a Phase 2 trial for its use in Charcot-Marie-Tooth disease. It is hoped to help patients who have muscle weakness causing reduced mobility and foot drop as a result of the condition.

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