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FDA Okays Marathon's Emflaza for DMD

FDA Okays Marathon's Emflaza for DMD

The drug was never sold in the US 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.

With FDA approval, Marathon now has exclusive rights to sell the drug in the U.S. for the next seven years, even though it has been available as a generic in other countries. 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 US; Marathon CFO Babar Ghias tells the Washington Post the net price will be $54,000 after rebates and discounts.

FDA approval of Emflaza marks a third major proof point of MDA's research program in recent months.

More news: Former French President Nicolas Sarkozy to stand trial over campaign financing

Marathon Pharmaceutical also sought approval of deflazacort as an "orphan drug", which is a special designation that provides incentives to assist and encourage the development of medicines for rare diseases. But given that the FDA approval will end the accepted practice of importing deflazacort into the USA for DMD treatment, it seems likely that at least some patients' costs could increase. 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. But deflazacort (brand named Emflaza) is thought by some to be most the effective and have the mildest side effects. Their ages ranged between 5 and 15 at the beginning of the trial. At Week 12, patients in the Emflaza group had improvements in clinical assessment of muscle strength across a number of muscles vs. patients taking placebo. Average muscle strength remained stable through the 52 weeks of the study for those on the corticosteroid, the agency said. Some studies have shown that patients on deflazacort tend to lose the ability to walk later that the ones who are not treated. 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.

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.

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