Adds background in paragraph 3, further details in paragraph 6, CEO quote in paragraph 7 and analyst comment in paragraph 8
By Christy Santhosh and Kamal Choudhury
March 28 (Reuters) - PTC Therapeutics PTCT.O said on Friday its inherited progressive muscle-wasting disorder drug will no longer be available for sale in the EU as the European Commission has decided not to renew its marketing authorization following a prolonged review.
The EC's decision backs the negative opinion on the drug, Translarna, given by the EU health regulator's advisory panel in 2023, which was later reaffirmed in January last year.
In May 2024, the EU regulator asked the panel to re-evaluate the treatment and to "further consider the totality of evidence, including data from patient registries and real-world evidence."
Translarna had received conditional approval in the European Union in 2014, making it the only available treatment for Duchenne muscular dystrophy (DMD) expressing "nonsense mutation", which prematurely ends the translation of a gene into a protein.
The drug is designed to boost production of a protein called dystrophin, the absence of which causes muscle weakening in patients with DMD.
PTC said the EC indicated that individual countries within the European Union can leverage certain clauses within the EU Directive to allow continued use of the drug.
"We look forward to working on a country-by-country basis to provide commercial drug where possible," said CEO Matthew Klein.
The company believes about 25% to 35% of EU Translarna revenue could be maintained through various measures, according to RBC Capital Markets analyst Brian Abrahams.
Translarna is not yet approved in the U.S. and was rejected by the Food and Drug Administration in 2016 and 2017, after the agency sought an additional trial to "provide substantial evidence of effectiveness."
Shares of the New Jersey-based company fell 1.4% in morning trading.
(Reporting by Christy Santhosh and Kamal Choudhury in Bengaluru; Editing by Shailesh Kuber)
((Christy.Santhosh@thomsonreuters.com))
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