FDA starts delivering on rare disease promises in genetic musculoskeletal disorders

FDA’s new leadership is delivering on its pledge to create viable pathways for rare disease drug development, at least for rare genetic musculoskeletal disorders.

On Monday, Avidity Biosciences Inc. (NASDAQ:RNA) reported biomarker and efficacy data from the Phase I/II FORTITUDE trial of delpacibart braxlosiran (del-brax) to treat facioscapulohumeral muscular dystrophy (FSHD). The biotech also announced that the accelerated approval pathway in the U.S. “is open” for del-brax. The confirmatory Phase III FORWARD study is enrolling patients, and a biomarker readout that could support accelerated approval is scheduled for next year...