Update on Translarna™ (ataluren) Application for Label Expansion

SOUTH PLAINFIELD, N.J., June 28, 2019 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has completed their review of a proposed indication extension of Translarna™ (ataluren) for the treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) who are non-ambulatory. While the CHMP adopted a negative opinion of the extension, PTC was informed by EMA representatives that the European Public Assessment Report (EPAR) will be updated to clarify that patients who start Translarna while ambulatory are not required to discontinue treatment after loss of ambulation.

«While we are disappointed with the current outcome of the label expansion procedure and its impact on non-ambulatory patients with nonsense mutation Duchenne Muscular Dystrophy, we are pleased that patients on Translarna can remain on treatment after loss of ambulation,» said Marcio Souza, Chief Operating Officer, PTC Therapeutics, Inc. «We remain committed to work with the CHMP to clarify the open questions and are

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Liz

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