FDA clears Marathon Pharmaceuticals' Emflaza for Duchenne muscular dystrophy

The FDA approved Marathon Pharmaceuticals' Emflaza (deflazacort) to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD). The agency said that this is the first clearance of a corticosteroid to treat DMD, noting that the drug works by decreasing inflammation and reducing the activity of the immune system.

"This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy," remarked Billy Dunn, director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research. Last year, the agency authorised Sarepta Therapeutics' Exondys 51 (eteplirsen) for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.

The approval of Emflaza was based on a study that included 196 male patients who were 5 to 15 years old at the beginning of the trial with documented mutation of the dystrophin gene and onset of weakness before age 5. Results showed that at week 12, those taking Emflaza had improvements in a clinical assessment of muscle strength across a number of muscles compared to those taking a placebo. In another study with 29 male patients that lasted 104 weeks, Emflaza demonstrated a numerical advantage over placebo on an assessment of average muscle strength.

FirstWord Reports: Providing insight, analysis and expert opinion on important Pharma trends and challenging issues <Click here>

"For the first time, patients in the US with Duchenne will have widespread access to an FDA approved medicine that is indicated for all genetic forms of the condition," commented Timothy M. Cunniff, executive vice president of R&D at Marathon. The company indicated that Emflaza will carry a list price of $89 000 a year, although the drugmaker said it provide a "robust" assistance programme for patients who are not covered by insurance.

To read more Top Story articles, click here.