FDA Approves Emflaza for Duchenne Muscular Dystrophy

Today, the FDA approved Emflaza (deflazacort) tablets and oral suspension for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years or older.

Emflaza is a corticosteroid commonly used to treat DMD in other countries, but this is the first FDA approval of any corticosteroid to treat DMD, according to an FDA press release. Furthermore it’s the first approval of deflazacort in the United States for any use.

“This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy,” said Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “We hope that this treatment option will benefit many patients with DMD.”

The approval was based on a clinical study that included 196 male patients aged 4 to 15 years at the start of the trial. Furthermore, patients had documented mutation of the dystrophin gene and onset of weakness before the age of 5.

A week 12, study participants administered Emflaza demonstrated improvements during a clinical assessment of muscle strength across a number of muscles compared with patients taking a placebo, according to the release. Overall stability in average muscle strength was maintained through the end of the study at week 52 in patients treated with Emflaza.

In a separate trial lasting 104 weeks that included 29 male patients, Emflaza showed a numerical advantage on an assessment of average muscle strength compared with placebo. Although the study was not controlled for multiple comparisons, patients administered Emflaza appeared to lose the ability to walk later than those treated with placebo, the release reported.

The most common adverse events (AEs) for Emflaza are facial puffiness, weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency, unwanted hair growth, and central obesity.

Less common AEs include issues with endocrine function, increased susceptibility to infection, elevation in blood pressure, risk of gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decrease in bone density, and vision problems.

DMD is a rare genetic disorder that results in muscle deterioration, and is caused by an absence of dystrophin. Patients with DMD progressively lose the ability to carry out everyday activities on their own, and often require a wheelchair by the time they reach their early teens. Patients typically die from the disease in their 20s or 30s due to heart and respiratory conditions.