Medical

Marathon stokes drug pricing flames after DMD approval

Marathon stokes drug pricing flames after DMD approval

Even though the drug has been available as a generic in other countries for quite some time, the federal designation also gives Marathon exclusive rights to market the drug in the US for seven years. And, like other pharma companies that scoop up "orphan drugs" - meds used for diseases that don't affect more than 200,000 people nationwide - Marathon says the high cost is justified because of the money it has poured into R&D.

For the last two decades, patients have been buying deflazacort from other countries ever since clinical trials demonstrated its effectiveness for treating Duchenne muscular dystrophy. 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.

Marathon Pharmaceuticals, the manufacturer of the drug, got approval to classify deflazacort as an "orphan drug", a category specifically meant to encourage research of drugs for very rare diseases.

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. DMD occurs in about one of every 3,600 male infants worldwide.

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Marathon anticipates a smooth transition for patients ready getting deflazacort from overseas pharmacies. Patients will no longer be allowed to import the drug now that an FDA-approved product is available. Sarepta's drug treats a subset of DMD patients representing about 13 percent of the total. The drug isn't a cure, but it has been shown to improve muscle strength, the FDA said in a statement announcing the approval.

However, some companies have used these incentives to their advantage and created business models based on procuring older drugs and substantially raising their prices.

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. The most 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). In addition, although not statistically controlled for multiple comparisons, patients on deflazacort appeared to lose the ability to walk later than those treated with placebo.


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