Saga over EU approval of PTC’s DMD therapy Translarna rumbles on

2024-06-28

上市批准加速审批

PTC Therapeutics PTC Therapeutics said Friday that it plans to request another re-examination of its conditional marketing authorisation of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) after the European Medicines Agency upheld its negative stance. Shares in the company fell around 13% on the news.

The regulator’s Committee for Medicinal Products for Human Use (CHMP) looked again at the data supporting Translarna at the request of the European Commission. Specifically, the CHMP was asked to assess the totality of evidence, including data from patient registries and real-world evidence.

Shortcomings in analysis

However, the EMA said that the additional evidence was not sufficient for a positive opinion on Translarna. The CHMP noted that methods used to conduct a meta-analysis of three clinical studies had “several shortcomings,” with the results not convincing enough to outweigh the negative findings of the individual trials. The EMA added that “no firm conclusion” on the effectiveness of Translarna could also be drawn from real-world data.

Translarna initially received conditional approval in Europe a decade ago for treating nmDMD in ambulatory patients aged five and older, which was later extended to include children as young as two years old. However, last year, while evaluating the annual renewal of Translarna’s marketing authorisation, the CHMP adopted a negative opinion, which PTC appealed.

The regulator then reaffirmed its recommendation against Translarna in January, although the European Commission decided against adopting the decision. PTC said Friday that the marketing authorisation for Translarna remains in effect, with the therapy expected to remain on the market through the end of 2024 even if the negative opinion is maintained and ratified by the European Commission.

‘Adds risk’ to US path

Commenting on the news, RBC Capital Markets analyst Brian Abrahams said the CHMP’s opinion will now go directly to the European Commission for a "final, legally binding decision.” Abrahams added that the regulator’s negative stance “perpetuates the uncertainty around Translarna in Europe and potentially adds risk to its path forward in the US."

In March, PTC outlined plans to re-submit a marketing application for Translarna to the FDA by mid-year, following feedback from the agency. The FDA rejected an earlier filing in 2017, saying at the time that an additional adequate and well-controlled trial would be needed to confirm the treatment's benefit.

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