Health regulators have given their approval for the drug deflazacort for patients suffering from Duchenne muscular dystrophy.
Available Overseas For $1,200
The steroid treatment has long been available outside United States but it was only last week that it received the green light from the U.S. Food and Drug Administration for Duchenne muscular dystrophy, a rare and incurable disease that most often affects boys and typically leads to death by the time the patient reaches his 30’s.
Families of sufferers used to import the drug from abroad at a cost of about $1,200 per year. Although the drug would now become more accessible with regulators’ approval, it comes with a literal high cost as it would be sold at a much higher price.
Marathon Pharmaceuticals will offer the drug at a cost of about $89,000 per year, which is up to 70 times more expensive compared to its cost outside of the U.S.
Marathon’s chief financial officer Babar Ghias said the net price of the drug will be $54,000 after rebates and discounts but some of those who rely on the drug are concerned they can’t afford the cost if insurance won’t pay for it and if the deductibles are too high.
Deflazacort, which will be sold in the U.S. under the brand Emflaza starting March this year, is said to cause fewer side effects compared with prednisone, a non-FDA-approved drug that is also often prescribed for DMD.
Deflazacort As An Orphan Drug
Marathon sought approval for deflazacort as an “orphan drug,” medicines used for diseases that do not affect more than 200,000 people in the U.S. Because of the drug’s orphan designation, Marathon gets seven years of exclusive rights to sell the drug in the country regardless that it has long been available overseas.
Just like other pharmaceutical company that scoop up orphan drugs, Marathon said that the high cost of the drug is justified by the amount of money it has poured into research and development. Marathon had to invest money in new trials before health regulators would approve the drug’s sales in the U.S.
Marathon likewise got a valuable priority review voucher that it can use to get a future drug reviewed faster by health regulators, or which it can sell to another company for hundreds of millions of dollars.
Financial Gains Of Getting FDA Approval For Old Drugs
The special approval is supposedly to encourage the development of drugs for rare diseases but critics of old drugs that get sold at higher prices think drug companies are gaming the system and reaping financial rewards as a result. In recent years, companies that have gotten FDA approval for old or existing drugs for rare disease gained big financial rewards.
“Instead of making the price at a level that is reasonable for patients, they make it a very high price,” said Aaron Kesselheim, from Harvard Medical School. “It’s a marketing move and not really a public health solution.”
Because FDA has already green-lighted the drug to treat muscular dystrophy, U.S. consumers would not be legally able to buy it overseas at a cheaper price anymore.
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