RARE Daily

Santhera’s Agamree Approved in United Kingdom to Treat DMD

January 16, 2024

Rare Daily Staff

The U.K. Medicines and Healthcare products Regulatory Agency has approved Santhera Pharmaceuticals’ Agamree in the United Kingdom for the treatment of Duchenne muscular dystrophy in patients 4 years of age and older, independent of the underlying mutation and ambulatory status.

The MHRA, adopting the view of the European Medicines Agency, acknowledged clinically important safety benefits of Agamree with regards to maintaining normal bone metabolism, density and growth compared to standard of care corticosteroids, alongside similar efficacy.

“We have secured a third approval for Agamree to treat Duchenne from a major regulatory agency, after the U.S. FDA and the EU EMA, within a couple months,” said Shabir Hasham, chief medical officer of Santhera. “In addition to its anti-inflammatory efficacy, both the EMA and the MHRA recognize the benefits of treatment with Agamree for bone health and growth, underlining the favorable safety and tolerability profile of this novel medicine compared to conventional corticosteroids.”

Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, is a rare and life-threatening neuromuscular disorder characterized by progressive muscle dysfunction, ultimately leading to loss of ambulation, respiratory failure, and fatality. Current standard treatment for DMD involves corticosteroids, which often come with significant side effects.

Agamree (vamorolone) mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity and, as such, is considered a novel corticosteroid with dissociative properties in maintaining efficacy, with a better-tolerated side effect profile. This mechanism of action may allow vamorolone to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients with DMD.

The approval by the EC was based on data from the positive pivotal VISION-DMD study and three open label studies in which vamorolone was administered for a total treatment period of up to 30 months. In the pivotal VISION-DMD study, boys treated with vamorolone on average maintained growth similar to those treated with placebo, while those treated with prednisone on average experienced growth stunting. Patients who switched from prednisone to vamorolone after 24-weeks were, on average, able to resume growing in height over the remainder of the study.

Unlike corticosteroids, vamorolone did not result in a reduction of bone metabolism as measured by bone biomarkers, nor in a significant reduction of bone mineralization in the spine as measured by Dual Energy X-Ray Absorptiometry after 48 weeks in the clinical studies. In addition, patients who switched from a standard of care corticosteroid to Agamree maintained the efficacy benefit while recovering their growth and bone health. Santhera will continue to collect data to further characterize the long-term effectiveness and the broader safety differentiation of vamorolone.

Agamree has Orphan Drug status for DMD in the United States and in Europe, Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine status from the U.K. MHRA for DMD. Agamree is approved for use in the United States, the European Union, and the United Kingdom.

Photo: Shabir Hasham, chief medical officer of Santhera

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