Alnylam gets an adcomm for Onpattro treatment in ATTR amyloidosis

Alnylam Pharmaceuticals is hoping its latest round of data for Onpattro’s treatment of ATTR is enough to satisfy the FDA. But they’ll have to make it through their advisors first.

Alnylam announced Tuesday that the FDA has accepted a supplemental NDA for Onpattro, otherwise known as patisiran, to treat the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis, and will be holding an advisory committee to discuss the application. However, the date of the adcomm was not immediately disclosed.

The FDA has set a PDUFA date for later this year on Oct. 8. As per an Alnylam release, the FDA’s acceptance letter said there were no review issues.

So far, the FDA has Onpattro indicated to treat the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Alnylam is going forward based on a Phase III trial, dubbed APOLLO-B, which investigated the drug for the quality of life in patients with ATTR amyloidosis against a placebo after 12 months. The trial showed the results hit the primary endpoint. Using the six-minute walk test against the placebo, the drug had a difference of 14.7 meters, earning a p-value of p=0.0162.

The adverse events were either mild or moderate, with events related to treatment occurring in 5% of patients and 3% in the placebo group. These included infusion-related reactions, muscle spasms and arthralgia. Five deaths were also observed in the Onpattro group, with eight deaths in the placebo, according to Alnylam.

Rena Denoncourt

“The FDA’s acceptance of our sNDA for patisiran is a positive step forward as we work to bring patients with ATTR amyloidosis with cardiomyopathy a new treatment option that addresses the underlying cause of disease and has the potential to meaningfully improve functional capacity and quality of life,” said Rena Denoncourt, VP and TTR franchise lead at Alnylam, in a release.

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