PTC Therapeutics has filed a Marketing Authorisation Application (MAA) for the potential approval of a gene therapy treatment, PTC-AADC, the company has announced.
Last year the drug garnered positive results in treating aromatic l-amino acid decarboxylase (AADC) deficiency, with data from a trial demonstrating clinically meaningful and sustained improvements in motor, cognitive and language milestones.
The investigational gene therapy hit endpoints when evaluated in the ability to sit, walk, and talk and represents up to five years of follow up post-treatment.
Following the submission of the MMA to the European Medicines Agency (EMA), PTC says that it expects an opinion from the Committee for Medicinal Products for Human Use (CHMP) in the second half of 2020.
To date, 200 AADC patients have been identified, and the company allegedly anticipates that over 300 AADC patients will be identified by launch.
The news means that PTC is “on the verge of bringing the first commercial treatment for AADC deficiency patients, which is in line with our mission of bringing clinically differentiated treatments to patients with rare disorders" added Stuart Peltz, chief executive officer.
The update was announced at the 38th Annual J.P. Morgan Healthcare Conference, along side preliminary 2019 financial results and 2020 financial guidance.
PTC has also released a few company announcements, including news that its PTC-FA gene therapy for Friedreich ataxia is progressing and is anticipated to enter the clinic in the third quarter of 2020.