[DMD 듀센형]Ataluren 임상 유럽에서 조기 승인을받지 못함

유럽 기관 인해 조기 정지 코돈 (넌센스 돌연변이)를 이용해 치료하는 ataluren에 대한 조건부 승인을 부여하기를 거부했다. 

진행중인 3 상 시험을 완료하기 전에 환자에게 약물을 사용할 수 있도록 하는 조건부 승인입니다.

3 상 시험 연구는 기준에 맞는 참가자들에게 열려 있습니다.

진행중인 임상실험이 완료되기전 약품사용을 허가요청했었나보네요.

아래는 원문입니다.

The experimental drug ataluren, in development for Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by specific mutations in the gene for the dystrophin protein, will not receive conditional approval at this time from the European Medicines Agency (EMA). Ataluren's developer is requesting that the EMA re-examine the data.

PTC Therapeutics, a New Jersey biopharmaceutical company that has been developing ataluren (formerly called PTC124) with support from MDA, announced the decision made by the European regulatory agency in a Jan. 24, 2014, press release. The company filed the conditional marketing application with the EMA in December 2012.

The drug is now in a phase 3 trial of 220 boys with nonsense mutation-related DMD or BMD who are 7 to 16 years old and meet other study criteria. The trial remains open to new participants. Conditional approval would have allowed the company to make the drug available to patients in Europe prior to the completion of the phase 3 trial, although full approval would still have required completion of this trial.

Results of the phase 3 trial are required for approval of ataluren by the U.S. Food and Drug Administration (FDA), regardless of what the EMA's final decision.

"While we appreciate that this delay is a disappointment for families living with this disorder, we are encouraged by the detailed discussions we have had with the EMA through this application process," said Stuart Peltz, CEO of PTC Therapeutics. "We understood and communicated that there was a substantial risk that the EMA would not grant us conditional approval when we began this process 15 months ago. However, we pursued this approach because we believe ataluren has shown a clinically meaningful benefit for nonsense-mutation DMD patients in our trials, has been generally well tolerated, and should be made available as soon as possible."