The five-year data of vamorolone (marketed as Agamree) in patients with Duchenne muscular dystrophy (DMD) indicates it is as effective as the standard corticosteroids used to treat the disease, but has a better safety profile, according to a recent press release by Santhera Pharmaceuticals.
Corticosteroids (such as deflazacort and prednisone) are often administered as standard-of-care in DMD, but long-term use causes significant side effects that may be distressing to patients.
Vamarolone is a corticosteroid that works by binding to the same receptors as the glucocorticoids typically used to treat DMD, but it has specific mechanisms that are intended to protect against the side effects typically observed in glucocorticoid use.
Santhera Pharmaceuticals conducted a study in which patients with DMD between four and seven years of age who had never been prescribed corticosteroids were started on vamarolone. The median follow-up period was five years, with some patients receiving treatment for up to eight years.
Patients on vamarolone were found to have maintained motor function. Vamarolone was associated with a comparable time to loss of ambulation, meaning that its efficacy profile was similar to that of other corticosteroids. Changes in body mass index or weight, when matched for height, did not differ between patients on vamarolone compared to those on other corticosteroids.
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Notably, vamarolone usage demonstrated better safety and tolerability compared with standard corticosteroid therapy. Researchers observed that patients on vamarolone had lower rates of vertebral fractures and fewer cases of cataracts, while maintaining normal growth without stunting. There had been no new cases of glaucoma. Furthermore, there were no new safety concerns associated with vamarolone.
“We are truly delighted to offer the DMD community a corticosteroid treatment option that can provide long-term benefit and significantly reduce the occurrence of some of the most debilitating side effects that often lead to dose down-titration or discontinuation,” Professor Eugenio Mercuri from the Universita Cattolica del Sacro Cuore said.
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