PTCT PTC Therapeutics Inc

By Stephen Nakrosis

PTC Therapeutics on Tuesday offered an update on regulatory activities regarding its Translarna protein restoration therapy in both the U.S. and Europe.

After the bell, PTC said it had submitted a briefing document as part of a re-examination of the Committee for Medicinal Products for Human Use's negative opinion on "the renewal of the conditional marketing authorization of Translarna in Europe." The re-examination will focus only on the renewal of the existing conditional marketing authorization, PTC said, adding the procedure is expected to last until late January of next year.

PTC also said it recently had a Type C meeting with the U.S. Food and Drug Administration to talk about a potential path to resubmit a new drug application for Translarna. PTC said based on the discussion, FDA suggested the company request a follow-up meeting. PTC said it expects the meeting will happen in the first quarter of 2024.

Translarna, or ataluren, which was discovered and developed by PTC Therapeutics, is "a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation," the company said.

Translarna is licensed in countries including Great Britain, Northern Ireland and the European Economic Area to treat nonsense mutation Duchenne muscular dystrophy in certain patients. Ataluren is an investigational new drug in the U.S., PTC said.

Write to Stephen Nakrosis at stephen.nakrosis@wsj.com

(END) Dow Jones Newswires

December 05, 2023 17:09 ET (22:09 GMT)

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