The FDA has rejected a request from Amicus Therapeutics to seek accelerated approval of an experimental drug for Pompe disease, asking for more data.
The agency indicated to the drugmaker that the current clinical package is not sufficient to support Accelerated Approval.
Amicus received similar bad news back in June from the EMA, when seeking conditional approval for the same development program. The Scientific Advice Working Party (SAWP) specifically noted that the efficacy data for AT-GAA appeared “promising” but that the current clinical package was not sufficient for a Conditional Marketing Authorization Application at the time.
Amicus Therapeutics intends to continue to generate data to support further discussions on a potential pathway for Accelerated Approval with the FDA in 2019.
Read the press release