PTC Therapeutics Gets FDA Priority Review of Upstaza Gene Therapy

By Colin Kellaher

PTC Therapeutics has won U.S. Food and Drug Administration priority review for its application seeking approval of its Upstaza gene therapy for the treatment of AADC deficiency, a rare genetic disorder.

The Warren, N.J., biopharmaceutical company on Tuesday said the FDA set a target action date of Nov. 13 for a decision on Upstaza, which won European Union approval in 2022 as the first gene therapy for AADC deficiency, which typically causes severe disability and suffering from the first months of life.

The FDA grants priority review to medicines that have the potential to provide significant improvements in the treatment of a serious disease, and the designation shortens the review period.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

05-14-24 0812ET