Amvuttra - opinion on variation to marketing authorisation

Opinion

EMA has issued an opinion on a change to this medicine's authorisation.

vutrisiran

Opinion

On 25 April 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending a change to the terms of the marketing authorisation for the medicinal product Amvuttra. The marketing authorisation holder for this medicinal product is Alnylam Netherlands B.V.

The CHMP adopted an extension to an existing indication to include treatment of wild-type or hereditary transthyretin amyloidosis in adults with cardiomyopathy. The full indications for Amvuttra will therefore be as follows:¹

Amvuttra is indicated for the treatment of hereditary-mediated transthyretin amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN).

Amvuttra is indicated for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.

---

¹ New text in bold, removed text as strikethrough

CHMP post-authorisation summary of positive opinion for Amvuttra (II-15)

AdoptedReference Number: EMA/CHMP/137435/2025

Key facts

• Name of medicine:
  Amvuttra
• EMA product number:
  EMEA/H/C/005852
• Active substance:
  vutrisiran sodium
• International non-proprietary name (INN) or common name:
  vutrisiran
• Therapeutic area (MeSH):
  Amyloid Neuropathies, Familial
• Anatomical therapeutical chemical (ATC) code:
  N07XX18
• Additional monitoring:

  This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

• Orphan:

  This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

• Marketing authorisation holder:
  Alnylam Netherlands B.V.
• Date of opinion:
  25/04/2025
• Status:
  Positive