FDA Okays Marathon's Emflaza for DMD

Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system

The drug will be available in both oral suspension and tablets, and can be administered to patients 5 years of age and older. In recent years, companies like Valeant Pharmaceuticals International and Turing Pharmaceuticals LLC have come under intense scrutiny from regulators and Congress for price gouging and abusing the rules just for profits.

The Wall Street Journal reports the now-FDA-approved deflazacort will be offered by Marathon Pharmaceuticals for $89,000 or so a year, up to 70 times its cost outside the U.S.; Marathon CFO Babar Ghias tells the Washington Post the net price will be $54,000 after rebates and discounts.

A recently FDA-approved drug used in steroid treatments, which has been on the worldwide market for years, will be introduced in the United States at the inflated price of $89,000 a year. Patients will no longer be allowed to import the drug now that an FDA-approved product is available. Unlike etelirsen, the drug will be approved for all DMD patients age 5 years and older. Although many questions, including pricing, still need to be answered, Parent Project Muscular Dystrophy said in a statement that the government's approval will increase access to more families that desperately need the treatment. "This also seems to be another example of gaming the Orphan Drug Act, which was meant to try and encourage research into new therapeutic entities for people who have rare diseases - and it doesn't seem like this is that". Image credit: USA Today.$89,000 a year to treat the disease in the U.S.

The drug wasn't sold in the US mainly because no company thought it would be profitable enough to warrant the effort of seeking FDA approval. Twelve weeks into the study, patients, who were taking deflazacort, showed marked improvements in muscle strength compared to those were taking a placebo. By Week 52, patients in the Emflaza group maintained an overall stability in average muscle strength. Patients in the Emflaza arm seemed to lose their walking ability later than patients in the placebo arm; this was not statistically controlled for multiple comparisons.

The side effects caused by Emflaza are similar to those experienced with other corticosteroids. This is the ninth rare pediatric disease priority review voucher issued by the FDA since the program began.

Other side effects that are less common include problems with endocrine function, increased susceptibility to infection, elevation in blood pressure, risk of gastrointestinal perforation, serious skin rashes, behavioral and mood changes, diminished bone density, and vision problems such as cataracts.