The FDA has opted not to approve a treatment for DMD, ataluren from PTC Therapeutics. The FDA requires the PTC company to conduct yet another clinical trial in order to consider approval of the treatment. Stuart W. Peltz, CEO of PTC Therapeutics Inc., responded that a formal request called “dispute resolution” will be filed with the FDA next week.
This is the third time the FDA has rejected the PTC’s application. This last refusal arrives a month after AdCom, an advisory committee of experts and parents. Here is what Stuart W. Peltz, CEO of PTC, communicated to the DMD community:
“We are extremely disappointed for the Duchenne community and strongly disagree with the agency’s conclusions,” said Stuart W. Peltz, Ph.D., chief executive officer of PTC Therapeutics. “We believe that this decision fails to consider the benefit-risk of ataluren and the high unmet medical need. Therefore, we plan to file a formal dispute resolution request next week.”
-Stuart W. Peltz, CEO of PTC Therapeutics Inc.
PTC Therapeutics continues to distribute ataluren to the families
The CEO, also reassured families currently receiving treatment through several clinical trial extension programs that they will continue to receive ataluren, throughout the “dispute resolution” appeal. A final decision about the distribution will be made following the outcome of the dispute resolution process. Regarding distribution for Canadian families, there is no information to suggest that there will be any change.
For more information, please consult:
- Press release, PTC
- PTC filling protest
- FDA decision leaves family few options
- No charm PTC Duchenne drug rejected
- PharmaTimes
- Rare Disease Report
Also in the same subject :
- FDA ADVISORY COMMITTEE (ADCOM) HEARS THE VOICE OF THE DMD COMMUNITY
- ADCOM FOR TRANSLARNA (ATALUREN) BY THE FDA
- Translarna™ (ataluren)
#AtalurenNow #laforcedmd
