Genea Biocells, a San Diego-based biotech company focused on drug development to treat neuromuscular diseases, today announced that it has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its therapeutic candidate, GBC0905, for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
Genea Biocells’ lead scientist for the FSHD program, Amanda Rickard, said, “Our targeted therapeutic candidate GBC0905 potently suppresses DUX4 function, prevents the activation of DUX4 target genes, and protects affected skeletal muscle cells in a dose-dependent fashion and, importantly, without affecting normal myogenesis. Aberrant DUX4 expression is the underlying cause of the disease and results in muscle fiber death. The FSHD field generally believes that blocking DUX4 will be curative. We are looking forward to thorough and expeditious development of this much-needed therapy.”
This is great news for the FSHD community, as the FDA’s designation confers a number of advantages to companies’ drug development efforts, including tax credits for qualified clinical trials costs and seven years of market exclusivity upon FDA approval of the drug.
Genea is one of several biotechs, including Fulcrum Therapeutics, Facio, and Ultragenyx, that are pursuing small molecule drugs aimed at suppressing DUX4 to treat FSHD.