PTC Therapeutics' Upstaza Granted Marketing Authorization By UK's MHRA For Treating AADC Deficiency

(RTTNews) - PTC Therapeutics, Inc. (PTCT) said on Thursday that its one-time gene replacement therapy Upstaza has been granted authorization by the UK's Medicines and Healthcare products Regulatory Agency for the treatment of AADC deficiency.

AADC or aromatic L-amino acid decarboxylase deficiency is a rare, severe disorder that blocks motor development resulting in serious disability.

Upstaza, the first gene therapy infused directly into the brain for the treatment of AADC deficiency, is indicated for patients 18 months and older patients.

The drug already has marketing authorization in all 27 European Union member states, as well as Iceland, Norway, Liechtenstein, and Northern Ireland.