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Alnylam scraps plans for expanding Onpatto label after FDA rejects filing in ATTR-CM

2023-10-09

临床3期上市批准

Alnylam Pharmaceuticals will no longer seek expanded approval of Onpattro (patisiran) in the US for the treatment for cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM)transthyretin-mediated amyloidosis (ATTR-CM) after the FDA rejected its application. The company said Monday that the agency issued a complete response letter citing insufficient evidence of clinical meaningfulness in the indication.

Onpattro won FDA approval back in 2018 for patients with polyneuropathy due to hereditary ATTR, while an advisory panel recently voted 9 to 3 that the benefits of the RNAi therapeutic outweigh its risks as a treatment for ATTR-CM. However, ahead of the advisory committee meeting, FDA scientists cast doubt on the magnitude of effect produced by Onpattro – which is given intravenously every three weeks - in the Phase III APOLLO-B study.

Alnylam noted that the CRL did not identify any issues with respect to clinical safety, study conduct, drug quality or manufacturing. The company indicated that it will now focus on continued development of the RNAi therapeutic vutrisiran for ATTR-CM. The Phase III HELIOS-B study of vutrisiran – which is administered subcutaneously once every three months – is currently ongoing.

CEO Yvonne Greenstreet said “we remain confident in the HELIOS-B…study of vutrisiran and look forward to sharing top-line results in early 2024.” The executive added “beyond vutrisiran, we are excited about the potential for ALN-TTRsc04, which may allow for greater TTR knockdown and less frequent dosing.”

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