Healthy Living

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

ACE-083 is being researched in two different phases

Acceleron is researching this drug in two phases. So far, phase 1 has proven to be successful, and the company aims to release its data from the second and final phase in late 2019.

In the first phase, 6 groups of patients, or cohorts, with various degrees of FSHD were given different doses of ACE-083 or a placebo injection, in either the bicep or the tibialis anterior (TA), which is the large muscle in the lower leg. After a number of injections over a 12 week period, all patients who received the orphan drug saw an improvement in the strength and growth of their muscles. 

There were also no reports of any negative side effects from the injection, other than slight irritation in the site itself. If the results continue to reflect a positive outcome, then this drug could be on the market very soon! 

Phase 2 of the study will be more random and involve a double-blind trial.  At that time, researchers will also be reviewing the safety of the drug as well the recommended dose for each muscle that the disease affects. So far, as many as 56 new patients are expected to participate in the trial.