16 July 2018 - - Cambridge, Massachusetts-based serious and rare disease therapeutics developer Acceleron Pharma Inc. (NASDAQ: XLRN) has been granted orphan drug designation by the US FDA for ACE-083, the company's locally acting myostatin+ muscle agent, for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD), the company said.
ACE-083 is a locally-acting therapeutic candidate, based on the naturally-occurring protein follistatin, which utilizes the myostatin+ approach to inhibit multiple TGF-beta ligands.
It is designed to have a concentrated effect along targeted muscles to maximize growth and strength selectively in the muscles into which the drug is administered.
Acceleron has developed ACE-083 for disorders such as Charcot-Marie-Tooth disease and facioscapulohumeral muscular dystrophy, in which improved muscle strength in target muscles may provide a clinical benefit and enhance quality of life. The product is currently being evaluated in phase 2 trials for each of these disorders.
FSHD is a rare genetic muscle disorder for which there are currently no approved treatments. The primary clinical presentation of FSHD is debilitating skeletal muscle weakness and loss.
The symptoms of FSHD develop in a descending pattern, beginning with the face and upper body and progressing to the lower body.
Acceleron is focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases.
Its research platform has generated four therapeutic candidates that are currently in clinical trials.