Treatment for Facioscapulohumeral Muscular Dystrophy Granted Orphan Drug Designation

Officials with the FDA have granted GBC0905, for the treatment of facioscapulohumeral muscular dystrophy (FSHD), an Orphan Drug Designation,

Genea Biocells announced in a press release.

FSHD, which affects between 4 and 10 per 100,000 people, is caused

by the abnormal expression of the DUX4

gene, which allows the DUX4 protein to be produced in cells and tissues where it is not typically present. Expression of the protein causes muscle weakness and atrophy that typically affects muscles in the face, shoulder girdle, and upper arms, though it can also affect muscles of the abdominal wall, hip, and thigh.

GBC0905, a small-molecule drug, suppresses DUX4

function and protects skeletal muscle cells from muscle fiber death. Amanda Rickard, lead scientist of Genea Biocells’ FSHD program, said in a statement that the company expects the drug to be curative.

Click to continue reading on The American Journal of Managed Care.