PTC Therapeutics Gives Updates on Translarna Regulatory Activities
05 Dicembre 2023 - 11:24PM
Dow Jones News
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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