Vamorolone
The question
To ask the Secretary of State for Health and Social Care, how many patients have accessed Vamorolone since it was given NICE approval for Duchenne Muscular Dystrophy; what assessment he has made of the adequacy of availability of Vamorolone to patients with DMD in that period; and whether the NHS had fully implemented the NICE recommendation by the April 2025 deadline.
Answered by Karin Smyth
The National Institute for Health and Care Excellence (NICE) published its technology appraisal, titled Vamorolone for treating Duchenne muscular dystrophy in people 4 years and over, on 16 January 2025. NHS England routinely commissioned vamorolone in line with the guidance, from 16 April 2025.
When a patient is started on vamorolone, the prescribing clinician submits an electronic prior approval form, confirming that NICE’s recommendation criteria are met. NICE’s costing report, included in the technology appraisal, suggested that 1,390 people expected to receive vamorolone in 2025/26. The NICE’s technology appraisal is available at the following link:
There is only 10 full weeks of data available since routine commissioning commenced in mid-April, and over this period there have been 32 prior approval forms submitted. Whilst submission of a form is not confirmation that treatment has begun, this is used as a proxy indication for the number of patients starting treatment.
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