Emflaza Approved for Duchenne Muscular Dystrophy

The price is 70 times higher than the drug's price in the United Kingdom according to DMD advocates. To compare, patients can snag the treatment for less than $1 through online pharmacies.

The company says it is "committed to working closely with payers to meet the patient and payer needs for acceptable access to Emflaza", including free access to the drug for patients without insurance. Simply put, it's because the drug has never been approved in the USA despite widespread availability to treat other inflammatory conditions like arthritis in dozens of other nations. With increasing number of DMD patients expected to go for a treatment, drug sales for 2017 were estimated at $138 million.

The company asked the FDA for approval of deflazacort as an orphan drug, which is a special pathway to approval that is meant to encourage more development of treatments for rare diseases.

A physical therapist treats a patient with DMD.

In a press release, Marathon mentioned significant investments the company made in research and development to bring the drug through the approval process. And until the approval of deflazacort, the price for it overseas was much lower.

But Marathon Pharmaceuticals (a member of the PhRMA trade group) finally got it FDA-approved Thursday under an "orphan drug" status, which is for drugs that treat rare diseases.

Deflazacort is the usual treatment for DMD across the world, but it is not a cure. But U.S. patients have been importing it from foreign countries since the 1990s after clinical trials showed its potential to reduce inflammation with fewer side effects than another steroid. Their ages ranged between 5 and 15 at the beginning of the trial. In general, the muscle strength was maintained among those taking the drug until the end of the 52-week study. 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 adverse events (AEs) associated with deflazacort are similar to those caused by other corticosteroids.

Common side effects include facial puffiness (Cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency (pollakiuria), unwanted hair growth (hirsutism), and excessive fat around the stomach (central obesity). It can also lead to a reduction in the density of the bones.