FDA New Drug and Biologic Approvals -- 2017 Year-in-Review

Mary L Windle, PharmD

Disclosures

January 11, 2018

In This Article

Emflaza (deflazacort)

Indication:

Duchenne muscular dystrophy (DMD) in adults and children aged 5 years or older.

Mechanism:

Glucocorticoid; oxazoline derivative of prednisolone. The precise mechanism by which deflazacort exerts its therapeutic effects in patients with DMD is unknown.

Dosage:

0.9 mg/kg/day PO once daily.

Approval:

Approval was based on a 52-wk study in boys aged 5-15 yr (n=196) with DMD. Deflazacort provided similar improvement in muscle strength compared with prednisone, but with less weight gain.

Reference:

Griggs RC, et al. Efficacy and safety of deflazacort versus prednisone and placebo for Duchenne muscular dystrophy. Neurology. 2016 Nov 15;87(20):2123-131.

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