Santhera Pharmaceuticals’ Agamree (vamorolone) has been accepted by the Scottish Medicines Consortium (SMC) to treat Duchenne muscular dystrophy (DMD) in patients aged four years and older.
The decision, which allows patients with the rare muscle-wasting disorder to access Agamree on the NHS in Scotland, comes one month after the National Institute for Health and Care Excellence (NICE) recommended the drug for use on the NHS in England, Wales and Northern Ireland.
Estimated to affect 2,500 males in the UK at any one time, DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles.
The disease causes progressive muscle degeneration and weakness, and its major milestones are the loss of ambulation and self-feeding, the start of assisted ventilation, and the development of cardiomyopathy.
Agamree is a dissociative anti-inflammatory drug that works in a similar way to existing corticosteroids, the current standard of care for children and adolescents with DMD, but without the same safety concerns.
The SMC’s decision follows the Medicines and Healthcare products Regulatory Agency’s approval of the therapy in January 2024, which was supported by results from the pivotal VISION-DMD study and three open-label studies.
In VISION-DMD, patients treated with Agamree on average maintained growth similar to those treated with placebo, while those treated with the corticosteroid prednisone on average experienced growth stunting.
The trial also showed that patients who switched from prednisone to Agamree after 24 weeks were on average able to resume growing in height over the remainder of the study.
Additionally, unlike corticosteroids, Agamree did not result in a reduction of bone metabolism or a significant reduction in bone mineralisation in the spine after 48 weeks in the clinical studies.
Commenting on the latest authorisation for the therapy, Santhera’s chief executive officer, Dario Eklund, said: “I am delighted that the team has secured this latest approval, which will ensure Scottish patients can benefit from this important treatment for DMD.
“We will work closely with NHS Scotland, along with all the key regulatory and health authorities in all our approved markets, to ensure patients have access to Agamree.”