Access to treatments

Vamorolone not recommended by NICE

Today, the National Institute for Health and Care Excellence (NICE) published its decision to not approve a treatment for Duchenne muscular dystrophy (DMD) called vamorolone. Vamorolone, also known by the brand name Agamree, was meant to be an alternative to conventional steroids such as prednisone or deflazacort.

MHRA approval

Vamorolone was approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in January 2024, and NICE’s decision to not fund it is about the cost effectiveness of the treatment.

In its provisional judgement to not recommend the drug for the NHS, NICE said:

Vamorolone is an effective treatment for DMD, but its relative effectiveness compared with other corticosteroids was highly uncertain… [and] there was not enough evidence to conclude that vamorolone is a cost-effective treatment option. 

What happens next? 

NICE’s decision is still provisional, and you can read the draft guidance here. A consultation is currently open on the draft guidance on NICE’s until 24 April 2024. NICE invites comments from the stakeholders for this evaluation, and from the public.

Duchenne UK want effective treatments for DMD, at prices that are affordable for our health system, with the system being flexible on price to take into account the complexity of DMD and the cost of developing medicines for our rare disease. As formal consultees, we will be commenting on the draft guidance, and we will share our thoughts on this draft guidance, and next steps after this news, with the community. 

NICE plans to reconsider its draft guidance at a meeting on Tuesday 7 May 2024, after which it will publish its final draft guidance (FDG). There will still be an opportunity to appeal the FDG before it is published as official NICE guidance. 

Why was NICE appraising vamorolone?

NICE is a body of the Department of Health and Social Care, and is responsible for assessing whether drugs should be made available through the NHS in England. Legally, the NHS in England must provide all treatments approved by NICE. Health matters are devolved, but Wales commits to following NICE technology appraisal guidance too, and Scotland and Northern Ireland also take NICE recommendations into account – though not automatically. 

NICE assesses the cost and clinical effectiveness of treatments through a process they call a ‘technology appraisal’, and then they produce ‘technology appraisal guidance’. Vamorolone was assessed through the single technology appraisal (STA) route, and you can find out more about the appraisals process here. 

What is vamorolone?

Vamorolone was created as an alternative to existing corticosteroids, also called glucocorticoids, which are routinely used in the treatment of DMD as they can reduce muscle inflammation and maintain muscle strength. 

Steroid treatment has several negative side effects, and vamorolone was developed to keep or improve the positives (efficacy profile) in comparison to existing steroids, but with the aim of showing fewer negatives (side-effects). Vamorolone’s success at achieving these aims is one of the features the NICE committee appraised. 

Vamorolone was developed by a US-based company called ReveraGen BioPharma Inc, though in 2020 Santhera Pharmaceuticals acquired the worldwide rights to the drug. Santhera is the company taking vamorolone through NICE. 

Our role in developing vamorolone

We played a major role in the development of vamorolone. In 2015, Duchenne UK, Joining Jack, and Duchenne Research Fund invested £750,000 towards a phase 1 clinical trial in boys with DMD, which then enabled ReveraGen to win a £6 million grant from Europe’s Horizon 2020. Our DMD Hub enabled swift recruitment to a phase 2 clinical trial in the UK, and Santhera has been engaging with us ever since to help secure access for patients in the UK. 

Published on 28 March 2024

Share this article