FDA Rejects Alnylam Pharmaceuticals’ Request for Expanded Approval of Onpattro for ATTR-CM

The U.S. Food and Drug Administration (FDA) has recently declined Alnylam Pharmaceuticals’ request for expanded approval of Onpattro, a drug that treats transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM). The FDA cited insufficient evidence of clinical meaningfulness for its decision.

Alnylam announced on Monday that they will no longer pursue the expanded indication after receiving a complete response letter from the FDA, indicating that the agency will not approve the application in its current form.

The FDA found that Alnylam, based in Cambridge, Mass., had not established the clinical meaningfulness of Onpattro’s treatment effects for the cardiomyopathy of ATTR amyloidosis. While an FDA advisory committee previously voted in favor of the benefits of Onpattro outweighing its risks for the treatment of this condition, they also suggested that the drug would be most useful for patients who experience worsening symptoms while receiving Pfizer’s tafamidis franchise of Vyndaqel and Vyndamax. Some Wall Street analysts had predicted that the FDA’s final decision would be uncertain.

It is important to note that the FDA did not raise any concerns related to clinical safety, drug quality, or manufacturing. This decision has no impact on Onpattro’s approved indication for the polyneuropathy of hereditary ATTR amyloidosis in adults.

Trading in shares of Alnylam, which closed at $176.92 on Friday, was temporarily halted premarket on Monday.

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