The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has approved Agamree® (vamorolone) as a safer alternative to corticosteroids in treating Duchenne muscular dystrophy (DMD). Developed by Santhera Pharmaceuticals, Agamree is recognized not only for its anti-inflammatory efficacy but also for its positive impact on bone health and growth, acknowledged by both the European Medicines Agency (EMA) and the MHRA.
This approval marks a significant milestone as Agamree becomes the first fully approved medicinal product for Duchenne in the EU and UK and the first treatment approved for DMD across the EU, UK, and the US. The drug is indicated for patients aged four and over, regardless of the underlying mutation and ambulatory status.
Dr. Shabir Hasham, Chief Medical Officer of Santhera Pharmaceuticals, highlighted the unique benefits of Agamree, emphasizing its role in bone health and growth. The approval in the UK sets the stage for Agamree to become a groundbreaking drug for the Duchenne community, offering a tailored solution for all individuals with Duchenne muscular dystrophy.
First EU, UK, and US-Approved Treatment for Duchenne Muscular Dystrophy
Vamorolone, the active ingredient in Agamree, binds to the same receptor as glucocorticoids but modifies downstream activity. Notably, previous research, including a 2022 study published in JAMA Neurology, suggests that vamorolone has the potential to separate efficacy from steroid safety concerns.
The European Commission’s approval of Agamree was based on comprehensive data from the VISION-DMD study and three other studies, showcasing the reported safety benefits of vamorolone. In the VISION-DMD study, boys treated with vamorolone maintained growth compared to those on a placebo, while prednisone users experienced growth stunting.
Vamorolone’s Distinctive Safety Profile: Preserving Bone Health in Duchenne Muscular Dystrophy Treatment
Importantly, vamorolone did not result in a reduction of bone metabolism, setting it apart from traditional corticosteroids. Unlike corticosteroids, vamorolone did not lead to a significant reduction in bone mineralization in the spine after 48 weeks, providing an added safety advantage.
Santhera Pharmaceuticals is now focused on making Agamree available to UK patients in the second half of 2024, pending the completion of pricing reviews by the National Institute for Health and Care Excellence (NICE). The initial European launch is planned for Germany in the first quarter of the year, signaling a positive step toward improving treatment options and enhancing the lives of those affected by Duchenne muscular dystrophy.
Resource: European Pharmaceutical Review, January 16, 2024
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