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FDA approves costly drug to fight Duchenne Muscular Dystrophy

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In an interview, Marathon CFO Babar Ghias noted the company will likely receive much less in net revenue than the list price, after providing discounts to government insurers and financial support to patients who can not afford the medication.

Corticosteroids are a common treatment option for DMD around the world but this is the first time that the FDA has approved any drug in the class to treat the condition and also the first time that Emflaza was approved for any kind of use. Marathon said the research and development costs necessary to make that happen justify the high price-tag of the drug.

Emplaza is a corticosteroid that reduces activity within the immune system and decreases inflammation. The drug, deflazacort, can help to treat patients ages 5 years and above that suffers from the disastrous and deadly disease Duchenne muscular dystrophy (DMD).

DMD is the most well-known kind of muscular dystrophy that caused by an absence of dystrophin, a protein that keeps muscle cells in place. The top 100 orphan drugs in the US cost an average of $111,820 a year per patient in 2014, according to a report by Evaluate, a market research firm.

Marathon Pharma's chiefe financial officer Babar Ghias told reporters that the net price of the drug will be $54,000 after some rebates and discounts.

Sen. Bernie Sanders (D-VT) and Rep. Elijah Cummings (D-MD) on Monday wrote to Illinois-based Marathon Pharmaceuticals CEO Jeffrey Aronin, calling the company's plan to charge $89,000 annually for its recently approved treatment "outrageous".

"Marathon's apparent abuse of government-granted exclusivity periods and incentives to sell what should be a widely available drug for $89,000 a year is unconscionable", Sanders and Cummings wrote in the letter.

Sanders and Cummings accused the company of taking advantage of the FDA's orphan drug program, which will give it seven years of exclusive sales rights on the treatment.

Clinical studies of the drug deflazacort showed improvement in muscle strength compare to those who took a placebo. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated patients. The agency granted fast track designation and priority review to the application.

The sponsor is receiving a rare pediatric disease priority review voucher under a program meant to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. Other side effects include elevated blood pressure, mood changes, infection, and serious skin rashes.

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