STAT+: Avidity, FDA reach agreement on accelerated filing of rare disease drug
A new agreement between the FDA and Avidity Biosciences could further assuage industry concerns about the agency’s stance on rare disease therapies.
Avidity Biosciences reached an agreement with the Food and Drug Administration to seek accelerated approval of a new treatment for an inherited muscle-weakening disease, the company said Monday.
The FDA’s willingness to consider an early approval for the Avidity drug should further assuage concerns from the biotech industry and investors about the agency’s stance on rare disease therapies in the face of staffing cuts and new leadership.
The San Diego-based company is developing its drug, called del-brax, to treat facioscapulohumeral muscular dystrophy, or FSHD, a genetic disease that causes muscles in the upper and lower body to weaken and lose function over time. Del-brax works by silencing an aberrant gene called double homeobox 4, or DUX4, that leads to high levels of a muscle-toxic protein by the same name.
