FDA Approves Emflaza for Duchenne Muscular Dystrophy

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The drug was never sold in the USA because companies didn't think it would be profitable enough, The Wall Street Journal reported, but some families have obtained the drug from online pharmacies overseas to help improve muscle strength for boys at least for a while.

Marathon Pharmaceuticals LLC's pricing of the drug, which has been available in Europe, is the latest example of a business model that has drawn ire from doctors, patients and legislators in recent years: cheaply acquiring older drugs and then drastically raising their prices. There is just one really big problem with the drug - It will cost patients $89,000 a year, an exorbitant price when compared to its cost in other countries around the world. Under that status, Marathon has exclusive rights to sell deflazacort in the United States for seven years.

The program is meant to encourage companies to develop drugs for rare diseases, but its use for the repurposing of existing drugs seems to some like an abuse of the program, the Post reported.

"Today we celebrate FDA approval of Emflaza as a treatment option for kids and adults living with Duchenne muscular dystrophy", said MDA President and CEO Steven M. Derks.

Marathon scored fast track designation, priority review, and orphan drug designation for deflazacort for DMD. Patients have been importing the drug since the 90s, but now they will have to pay 50 to 70 times what they used to pay if they want to get it in the U.S.

The news of deflazacort's approval, as well as its high sticker price, has parents of DMD children mostly excited. Their ages ranged between 5 and 15 at the beginning of the trial.

In another, 104-week trial with 29 male patients, deflazacort showed a "numerical advantage over placebo on an assessment of average muscle strength", according to the FDA press release, which also noted that patients on deflazacort "appeared to lose the ability to walk later than those treated with placebo", although this was not statistically controlled for multiple comparisons. Average muscle strength remained stable through the 52 weeks of the study for those on the corticosteroid, the agency said.

Though side effects caused by the drug have been reduced over the years, they remain similar to those resulting from other corticosteroids. Adverse effects associated with Emflaza include facial puffiness, weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency, unwanted hair growth, and excessive fat around the stomach. It can also lead to a reduction in the density of the bones.