PTC Therapeutics To Withdraw Its Conditionally Approved Rare Muscle Disorder Disorder Drug In Europe – PTC Therapeutics (NASDAQ:PTCT)

Thursday, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion following the re-examination procedure for the conditional marketing authorization of PTC Therapeutics Inc’s PTCT Translarna (ataluren).

“We are disappointed that the CHMP has maintained its negative opinion on the Translarna authorization, which will result in the withdrawal of a therapy for patients in Europe with nonsense mutation Duchenne Muscular Dystrophy that the data support is safe and effective,” said Matthew Klein, Chief Executive Officer of PTC Therapeutics. 

In September 2023, the CHMP gave a negative opinion on converting the conditional marketing authorization to full marketing authorization of Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy

Translarna received conditional approval in the European Union in 2014 to treat ambulatory (who can walk) children aged five years and older with Duchenne muscular dystrophy expressing nonsense mutation. 

Also Read: PTC Therapeutics Stock Tumbles: Company Stops Preclinical Gene Therapy Programs After Failed Friedreich’s Ataxia Study.

Translarna is an investigational new drug in the U.S. 

Following the company’s announcement of top-line results from the placebo-controlled trial of Study 041 in June 2022, the company submitted a meeting request to the FDA to clarify the regulatory pathway for a potential re-submission of a New Drug Application for Translarna.

The company has scheduled an additional Type C meeting with the FDA to review the totality of data collected to date, including dystrophin and other mechanistic data, as well as additional analyses that could support the benefit of Translarna.

The FDA provided initial written feedback that Study 041 does not provide substantial evidence of effectiveness to support an NDA re-submission.

In Q3, Translarna’s net product revenues were $69.0 million for the three months ended September 30, 2023, a decrease of 10% Y/Y.

Read Next: PTC Therapeutics’ Seizure Study Fails To Meet Primary Goal, Analyst Says Minimal Impact On Investment Thesis.

Price Action: PTCT shares traded 8.85% lower at $24.30 during the after-hours trading on Thursday.