PTC Therapeutics Gets CHMP Backing for Upstaza in AADC Deficiency
May 20 2022 - 8:54AM
Dow Jones News
By Colin Kellaher
PTC Therapeutics Inc. on Friday said the European Medicines
Agency's Committee for Medicinal Products for Human Use recommended
approval of its Upstaza gene-replacement therapy for the treatment
of aromatic L-amino acid decarboxylase, or AADC, deficiency.
The South Plainfield, N.J., pharmaceutical company said Upstaza,
if approved by the European Commission, would be the first
disease-modifying treatment for the rare genetic disorder for
patients 18 months and older and the first marketed gene therapy
directly infused into the brain.
The European Commission, which generally follows the CHMP's
advice, is expected to make a decision in about two months, PTC
said.
Write to Colin Kellaher at colin.kellaher@wsj.com
(END) Dow Jones Newswires
May 20, 2022 08:39 ET (12:39 GMT)
Copyright (c) 2022 Dow Jones & Company, Inc.
PTC Therapeutics (NASDAQ:PTCT)
Historical Stock Chart
From Mar 2024 to Apr 2024
PTC Therapeutics (NASDAQ:PTCT)
Historical Stock Chart
From Apr 2023 to Apr 2024
