Final NICE nod for Santhera’s Agamree

16 January 2025

The UK’s health technology assessor, the National Institute for Health and Care Excellence (NICE) has issued positive Final Guidance that recommends Agamree (vamorolone) for use in the National Health Service (NHS) in England, Wales and Northern Ireland for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older.

Swiss specialty pharma firm Santhera Pharmaceuticals (SIX: SANN), the developer of the drug, noted that this follows confirmation that no appeals were received against the Final Draft Guidance (FDG) recommendation announced in December. Following this, Santhera has already started launch preparations for Agamree in the UK and expect first sales in this quarter.

According to Santhera, Agamree is the first and only medicinal product for DMD to have received full approval in the European Union, USA, and UK. This recommendation follows the Medicines and Healthcare products Regulatory Agency’s (MHRA) approval of Agamree on January 11, 2024, which, alongside the European Medicines Agency (EMA), acknowledged clinically important tolerability benefits with regards to maintaining normal bone metabolism, density, and growth compared to standard of care corticosteroids, alongside similar efficacy from clinical trials.

This article is accessible to registered users, to continue reading please register for free.  A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Login to your account

Become a subscriber

 

£820

Or £77 per month

Subscribe Now
  • Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
  • Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
  • Daily roundup of key events in pharma and biotech.
  • Monthly in-depth briefings on Boardroom appointments and M&A news.
  • Choose from a cost-effective annual package or a flexible monthly subscription
The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK

Companies featured in this story

More ones to watch >


Today's issue

Company Spotlight





More Features in Pharmaceutical