Duchenne Muscular Dystrophy Treatment Emflaza Approved by FDA

MDA Celebrates News of FDA Approval of Emflaza for Treatment of Duchenne Muscular Dystrophy

The company said it would respond to the letter later Monday. With the recent approval, Emflaza hit the USA markets with a price tag of $89,000 a year, even when the company offers the same drug in Europe for a fraction of that cost.

Vermont Business Magazine Senator Bernie Sanders (I-Vermont) and Representative Elijah Cummings (D-MD), ranking member of the House Committee on Oversight and Government Reform, sent a letter to Marathon Pharmaceuticals Monday demanding answers about its plan to charge $89,000 per year for deflazacort, a drug that is widely available overseas for approximately $1,000 per year.

The practice has prompted congressional investigations and hearings into companies including Valeant Pharmaceuticals International Inc. and Turing Pharmaceuticals LLC, the firm formerly run by onetime hedge-fund manager Martin Shkreli.

It seems to be part of a trend of snapping up older drugs for not much money and then greatly increasing the price tag, forcing the patients to foot the bill.

Here is the statement from the FDA.

The U.S. Food and Drug Administration on Thursday approved a Northbrook pharmaceutical company's drug to treat a deadly form of childhood-onset muscular dystrophy - only the second FDA-approved drug for the disease and the first for everyone with the disorder.

As an immunosuppressant and anti-inflammatory, Emflaza was shown to slow loss of muscle function and strength, maintain respiratory and cardiac function, and reduce incidence of scoliosis in DMD patients.

Tracy also noted that while deflazacort is useful in DMD - as are other steroids, including prednisone, which is generic and has some efficacy evidence behind it - it is a purely symptomatic treatment, providing improved muscle strength. While the steroid is commonly used globally, the FDA's action represents the first approval of Deflazacort for use in the U.S. While the compound has been on the market for decades outside the country-cheaply imported to treat the deadly muscle-wasting disease-it hasn't before been formally approved by USA regulators, who granted it orphan drug designation.

There is no cure for Duschenne muscular dystrophy.

DMD is a rare, genetic disease caused by mutations in the dystrophen gene. DMD affects about one of every 3,600 male infants globally, according to the FDA. As the disease progresses, life-threatening heart and respiratory conditions can occur. Patient typically pass away in their 20s or 30s, but life expectancy may vary per patient depending on severity of DMD.

The drug's effectiveness was observed in a clinical study involving 196 male patients between the ages of 5 and 15.



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