FDA Okays Marathon's Emflaza for DMD

The big issue facing closely held Marathon will be how to price the drug.

Marlin, who has treated his children with imported deflazacort from the United Kingdom, is hopeful though that the FDA's decision will spark future drug approvals.

The Food and Drug Administration has approved emflaza (deflazacort) to treat patients with Duchenne Muscular Dystrophy, which is a rare genetic condition that leads to muscle deterioration and weakness. The bad news is that it comes with a high price of $89,000 a year. Unlike etelirsen, the drug will be approved for all DMD patients age 5 years and older.

Derks said, "Today we celebrate FDA approval of Emflaza (deflazacort's brand name) as a treatment option for kids and adults living with Duchenne muscular dystrophy ..."

The sponsor is receiving a rare pediatric disease priority review voucher under a program meant to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

The drug, Emflaza, known generically as deflazacort, belongs to a class of anti-inflammatory drugs known as corticosteroids that are frequently used to treat DMD and other conditions. It has been available for years in other countries, and the family of the individuals with Duchenne Muscular Dystrophy. FDA approval should also increase access to a drug that may be too expensive to buy outright for some, even in its generic form. However, the drug was never approved by the FDA as no companies thought it profitable enough to jump through the regulatory hoops necessary to sell it in the U.S.

At week 12, those on deflazacort had improvements in a clinical assessment of muscle strength across a number of muscles compared with those on placebo. By Week 52, patients in the Emflaza group maintained an overall stability in average muscle strength. In addition, although not statistically controlled for multiple comparisons, patients on deflazacort appeared to lose the ability to walk later than those treated with placebo. This is the ninth rare pediatric disease priority review voucher issued by the FDA since the program began.

Side effects associated with taking Emflaza are similar to that of other corticosteroids including, puffiness of the face, weight gain, an increased appetite, infection of the upper respiratory tract, frequent urination, cough, stomach fat, and unwanted hair growth.

As well, some patients on the drug can develop certain problems with endocrine functions, elevated blood pressure, risks of gastrointestinal perforation, skin rashes, cataracts, and susceptibility to infections.

  • Santos West