NICE Final Draft Guidance backs Alnylam’s Amvuttra

The UK subsidiary Alnylam  Pharmaceuticals (Nasdaq: ALNY) welcomed positive Final Draft Guidance issued by the National Institute for Clinical Health and care Excellence (NICE) has recommending the firm’s Amvuttra (vutrisiran) for eligible adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM) in England and Wales.

The health technology assessor’s final draft guidance is subject to appeal, with final guidance anticipated in December 2025.

Vutrisiran is the first and only long-acting disease modifying therapy (which can be self-administered every three months) to target the root cause of ATTR-CM. It delivers a rapid and sustained ‘knockdown’ of transthyretin (TTR) proteins – observed as early as six weeks after initiation of treatment – meaning it reduces the production of TTR proteins, including the unstable, misfolded proteins that can cause this life-threatening cardiac disease.