The National Institute for Health and Care Excellence (NICE) has recommended Santhera Pharmaceuticals’ Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD).
The drug has been recommended for use on the NHS in England, Wales and Northern Ireland to treat the rare muscle-wasting disorder in those aged four years and older, with approximately 1,700 patients expected to benefit from the endorsement in England alone.
Estimated to affect one in every 3,500 male births globally, DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles. Major milestones of the disease are the loss of ambulation and self-feeding, the start of assisted ventilation, and the development of cardiomyopathy.
Corticosteroids are used to reduce muscle inflammation and maintain muscle strength and function in DMD patients, but are associated with side effects including osteoporosis, reduced bone strength, increased risk of spinal fractures, and growth restriction.
Santhera’s Agamree offers an alternative to existing corticosteroids without the same safety concerns.
NICE’s final draft guidance follows the Medicines and Healthcare products Regulatory Agency’s approval of the therapy in January, which acknowledged clinically important safety benefits compared to standard-of-care corticosteroids, alongside similar efficacy from clinical trials.
Santhera said it will be working closely with the NHS to “ensure rapid patient access” to Agamree, adding that it is pursuing reimbursement for the drug via the Scottish Medicines Consortium to ensure access for patients in Scotland.
Helen Knight, director of medicines evaluation, NICE, said: “Given the significant burden this disease places on children with the condition and on their parents and carers, as well as the negative impact this can have on their quality of life, a treatment with the potential to reduce side effects associated with current treatments is to be welcomed.”
Also commenting on the agency’s decision, Dario Eklund, Santhera’s chief executive officer, said: “DMD is a devastating condition, the most common and severe form of muscular dystrophy, and patients urgently need improved treatment options.
“We are delighted that NICE has recommended Agamree for patients with DMD, and we are committed to working closely with the NHS to ensure patients can access Agamree as quickly as possible.”