Alnylam scores a first with FDA nod for Amvuttra in ATTR-CM

US RNAi therapeutics company Alnylam Pharmaceuticals (Nasdaq: ALNY) announced the US Food and Drug Administration (FDA) approval of the supplemental New Drug Application (sNDA) for its RNAi therapeutic, Amvuttra (vutrisiran), for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.

The approval expands the indication for Amvuttra, which now becomes the first and only therapeutic approved by the FDA for the treatment of ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. Alnylam’s shares were up 4.4% at $264.83 in pre-market activity today.

The approval was largely expected, though CVH/UHF reduction below that of acoramidis (noting usual caveats on cross-trial comparison) will be a headwind for Amvuttra, noted Leerink Partners analyst Dr Mani Foroohar. Alnylam announced it will maintain list price at $119,000/injection ($476,000 annually). While holding Pthe price at ~2x that of approved competitors will increase Street estimates, Dr Foroohar expects significant payer headwinds to commercial uptake (outside of academic centers with established buy-and-bill infrastructure and strong financial incentives to choose a more expensive Part B drug).