FDA approves muscular-dystrophy drug costing $89000 in the US

FDA approves muscular-dystrophy drug costing $89000 in the US”

Ghias defended the heavy price tag of the drug, saying the high cost was justified due to the big amount of money the company poured into research and development (R&D).

Prices for medications such as deflazacort, which are used to treat rare diseases and called orphan drugs, are often much higher than drugs for more common ailments.

While corticosteroids are the standard treatment commonly used globally to treat DMD, this is "the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use" in the US, the agency said. DMD commonly manifests in individuals without known family histories of the condition and mainly affects boys. This particular drug is meant to treat a disease that impacts about 12,000 boys each year in the United States. Since Marathon won an approval to treat a condition that afflicts fewer than 200,000 Americans per year and has a dearth of treatment options (rather than trying to become an also-ran in the saturated steroid therapy field), it has carte blanche over the drug's pricing.

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A USA Today report reveals the drug, a steroid known as deflazacort, will cost patients a whopping $89,000 per year, almost 70 times higher than buying the drug in other countries. It is the first corticosteroid approved by the FDA that is hopeful to help treat patients with DMD. The Wall Street Journal reports the now-FDA-approved deflazacort will be offered by Marathon Pharmaceuticals for $89,000 or so a year, up to 70 times its cost outside the U.S.; Marathon CFO Babar Ghias tells the Washington Post the net price will be $54,000 after rebates and discounts.

Patients with DMD typically do not live past their 20s or 30s, according to the FDA. Sarepta's drug treats a subset of DMD patients representing about 13% of the total.

Clinical studies of the drug deflazacort showed improvement in muscle strength compare to those who took a placebo. In general, the muscle strength was maintained among those taking the drug until the end of the 52-week study.

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Similar results were observed in another trial that lasted 104 weeks with 29 male patients but this experiment also showed that participants on deflazacort seemed to lose walking ability later than those given placebo. This is the ninth rare pediatric disease priority review voucher issued by the FDA since the program began.

As well, some patients on the drug can develop certain problems with endocrine functions, elevated blood pressure, risks of gastrointestinal perforation, skin rashes, cataracts, and susceptibility to infections.

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