Deflazacort in Duchenne muscular dystrophy: a profile of its use in the USA

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Funding: The preparation of this review was not supported by any external funding.

Conflicts of interest: K. McKeage and K.A. Lyseng-Williamson are employees of Adis/Springer, are responsible for the article content and declare no conflicts of interest

Additional information about this Adis Drug Review can be found here.

Abstract

Deflazacort (Emflaza™) is a derivative of prednisolone with immunomodulatory and anti-inflammatory properties. Overall, in the treatment of boys with Duchenne muscular dystrophy (DMD), deflazacort is as effective as prednisone, but may be better tolerated. In a randomized, double-blind, phase 3 trial in boys with DMD, deflazacort and prednisone both preserved muscle strength more effectively than placebo after 12 weeks of treatment, with improvements being maintained until study end at week 52. Both active treatments were also associated with improved motor function. In the real-word setting, treatment with corticosteroids for ≥ 1 year significantly delayed disease progression relative to no or < 1 month of corticosteroid treatment, with deflazacort showing some benefits over prednisone/prednisolone. Body weight gain-related events were less frequent with deflazacort than with prednisone, but stunted growth and the development of non-serious cataracts were more frequent. Access to the full article can be found here.

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