FDA Approves Muscular Dystrophy Drug, Will Cost Patients $89000

Zachary BarrettFeb 14, 2017

Deflazacort has been available outside the United States for decades.

A drug to treat muscular dystrophy will hit the US market with a price tag of $89,000 a year despite being available for decades in Europe at a fraction of that cost.

There's a new Duchenne muscular dystrophy (DMD) drug on the scene-at least, technically speaking. That pharmacy recently informed customers that it would stop shipping the drug to the United States after Marathon received FDA approval for its version.

The regulator gave a green light to Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with the rare genetic disorder, which causes progressive muscle deterioration and weakness in children, nearly always boys. Individuals with this condition lose little by little their ability to perform activities on their own, and they usually require the use of a wheelchair before their 20s. DMD occurs in about one of every 3,600 male infants worldwide.

The two lawmakers said they believe Marathon is abusing a program that grants companies seven years of market exclusivity to encourage research into new treatments for rare diseases.


Emflaza is marketed by Marathon Pharmaceuticals, headquartered in Northbrook, Illinois.

The drug wasn't previously sold in the USA because there was no company that would think it was profitable enough to warrant the effort of getting FDA approval. Competitor Sarepta Therapeutics has said its disease-modifying DMD drug, Exondys 51, will cost about $300,000 annually for the average patient in the U.S. Prednisone, another steroid, is frequently prescribed for the disease though it does not have official FDA approval. That drug, however, only helps about 13 percent of Duchenne patients, those with a specific genetic mutation, said Tim Cunniff, Marathon executive vice president for research and development.

How was the company able to get away with such an outrageous list price in America?

Deflazacort is the usual treatment for DMD across the world, but it is not a cure. At week 12, patients taking deflazacort had improvements in a clinical assessment of muscle strength across a number of muscles compared to those taking a placebo.

Emflaza side effects are similar to other corticosteroids, such as puffiness of the face, weight gain, increased appetite, infection of the upper respiratory tract, fequent urination, stomach fat, cough, and unwanted hair growth. In addition, patients who were given deflazacort lost the ability to walk later than those patients who were taking the placebo.

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