FDA’s August PDUFAs revisit rejections

FDA has at least 11 decisions due in August, including three involving gene and cell therapies or vaccines reviewed by CBER. Two of the three sponsors are seeking to overcome earlier regulatory setbacks under the agency’s new leadership, one of which suffered a setback at an FDA panel meeting on Wednesday.

Capricor Therapeutics Inc. (NASDAQ:CAPR) may be facing an uphill battle in the coming decision on deramiocel to treat cardiomyopathy in patients with Duchenne muscular dystrophy. Members of the Cellular, Tissue, and Gene Therapies Advisory Committee rebuffed the allogeneic cardiac-derived cell therapy, voting 3 to 9 on the lone voting question of efficacy in cardiomyopathy. Capricor received a complete response letter for the BLA last July requesting more clinical data; the company reported positive data in December that it hopes will support the application...