The US FDA (Food and Drug Administration) has approved Vamorolone for the treatment of Duchenne muscular dystrophy in children and adults aged 2 and over. It will be sold under the brand name Agamree and is expected to be available in the US in early 2024. This follows the announcement that the EMA has recommended the treatment for approval in Europe for those aged 4+.
Vamorolone is an alternative to corticosteroids. It was developed with the aim of maximising the benefit of steroids while minimising any side effects.
The Duchenne Research Fund played a significant role in the development of Vamorolone. Together with our friends at Duchenne UK and Joining Jack, we contributed to the funding of the $2.1 million Phase 1 clinical trial of Vamorolone, which enabled ReveraGen BioPharma to win a £6 million EU Horizon 2020 grant to continue its research into Phase 2 and beyond.
Corticosteroids are part of daily life for many boys and young men living with Duchenne, however the side effects can be significant. The approval of Vamorolone is an important step forward, and also marks the first time a treatment has been approved for all Duchenne patients regardless of mutation.
Vamorolone is now under review by the MHRA for approval in the UK, and will also be evaluated by NICE to determine if the treatment will be made available on the NHS.
Learn more about how our funding of Vamorolone has impacted the Duchenne arena.
Read Santhera’s press release about the approval.