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Deflazacort Approved for Duchenne Muscular Dystrophy

Kate Traynor

Kate TraynorNews Writer
News Center

FDA and Marathon Pharmaceuticals LLC on February 9 announced the approval of deflazacort oral tablets and oral suspension for the treatment of Duchenne muscular dystrophy in patients 5 years of age or older.

Deflazacort is the first corticosteroid approved by FDA for the treatment of Duchenne muscular dystrophy, a genetic disorder that affects 1 of every 3,600 infants worldwide, according to the agency.

Marathon stated that the drug will be available early this year under the brand name Emflaza and will be distributed by an unnamed “top specialty pharmacy.”

Access to the FDA-approved product is managed through the Emflazacares program. Prescribers must submit their patients’ clinical and insurance documentation to the program before initiating therapy.

In a placebo-controlled trial involving 196 male patients 5–15 years of age, treatment with deflazacort was associated with significant improvements in average muscle strength scores after 12 weeks of therapy. Each study participant had a mutation in the dystrophin gene, onset of muscle weakness before 5 years of age, and a history of elevated serum creatinine kinase levels.

The most frequently reported adverse events associated with the use of deflazacort in the study included facial puffiness, weight gain, increased appetite, upper respiratory tract infection, cough, high daytime urinary frequency, hirsutism, and central obesity. FDA stated that these adverse events are similar to those reported with the use of other corticosteroids.

The labeling (PDF) for deflazacort includes warnings about serious adverse events tied to corticosteroid use, including increased risk of infection, Cushing’s syndrome, hyperglycemia, and adverse changes in cardiovascular and kidney function.

The recommended dosage of deflazacort is approximately 0.9 mg/kg/day administered once daily. For patients taking the tablet form of the drug, the labeling advises using an appropriate combination of tablet strengths and rounding up the calculated dose to the closest achievable one.

Abrupt cessation of deflazacort in patients who have been taking the drug or another corticosteroid for more than a few days can result in fatal adrenal insufficiency. A gradual decrease in dosage should be used when discontinuing therapy, according to the labeling.

Deflazacort will be supplied as 6-mg tablets packaged 100 per bottle and 18-, 30-, and 36-mg tablets packaged 30 per bottle. The drug will also be available as a 13-mL oral suspension at a concentration of 22.75 mg/mL for addition to milk or a juice other than grapefruit juice.

All formulations of the drug should be stored at controlled room temperature. Unused suspension should be discarded one month after first opening the bottle. Each bottle comes with two 1-mL oral dispensers.

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