Access to treatments

Vamorolone considered by NICE committee

A technology appraisal committee (TAC) at the National Institute for Health and Care Excellence (NICE) considered vamorolone (also known under the brand name Agamree) today.

Approved by the MHRA

At the start of 2024, the Medicines and Healthcare products Regulatory Agency (MHRA) approved vamorolone for the treatment of Duchenne muscular dystrophy (DMD) in patients aged four years and older.

But people with DMD will not be able to access the treatment through the NHS until an assessment is made by NICE. The meeting today was a key milestone in that NICE assessment, and they plan to publish their final decision in May 2024.

What is the NICE committee, and what happened?

NICE’s TACs meet to consider treatments being assessed by NICE. Its recommendations are very important – patients have a right to treatments approved by NICE for use in the NHS, if their clinician believes it is appropriate for them.

TACs can only make certain recommendations and, for treatments like vamorolone, they are typically recommended, not recommended, or preliminary recommendation dependent on clarification.

TAC C met today to discuss vamorolone and DMD. It heard from selected experts, such as clinicians and parents to boys with DMD, and was shown a presentation about DMD and the treatment. The first part was held in public, and the second part held in private for discussion and deliberation.

You can find the page on NICE’s assessment of vamorolone on their website here.

What happens next?

TAC C did not make a decision today. They plan to publish their decision on 22 May 2024.

What is vamorolone?

Vamorolone was developed 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 is appraising.

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 5 March 2024

Share this article