as informed in Ghias said that the company carefully thought about pricing and came up with a price far lower than other rare disease drugs — in the bottom 10th percentile of rare disease drug prices.
An old steroid treatment, long available outside the United States, received approval this week for a rare disease that afflicts about 15,000 Americans.
The company sought approval for deflazacort as an “orphan drug,” a special approval pathway intended to encourage the development of drugs for very rare diseases.
For example, tetrabenazine, a drug that was available from abroad and used for years to treat the uncontrollable tremors of Huntington’s disease, was approved as an orphan drug in 2008.
In recent years, companies that have gotten old or existing drugs approved to treat rare diseases have reaped big financial rewards.
as declared in A Drug to treat muscular dystrophy will hit the U.S. market with a price tag of $89,000 a year despite being available for decades in Europe at a fraction of that cost.
The U.S. Food and Drug Administration on Thursday approved Marathon’s drug, a corticosteroid called deflazacort, to treat a rare type of muscular dystrophy that affects some 12,000 boys in the U.S., most of whom die in their 20s and 30s.
The Drug isn’t a cure, but it has been shown to improve muscle strength, the FDA said in a statement announcing the approval.
The practice has prompted congressional investigations and hearings into companies including Valeant Pharmaceuticals International Inc. and Turing Pharmaceuticals LLC, the firm formerly run by onetime hedge-fund manager Martin Shkreli.
The Drug wasn’t sold in the U.S. mainly because no company thought it would be profitable enough to warrant the effort of seeking FDA approval.
as declared in
Old drug gets an absurd price hike to $89,000 per year
However, the price hike underscores a problem with American pharmaceutical companies exploiting the orphan drug label to reap massive profits from medicine.
The practical cost will be negligible if you have appropriate health insurance, and Marathon contends that this is a low price for a rare disease drug.
FDA approval should also increase access to a drug that may be too expensive to buy outright for some, even in its generic form.
As helpful as the approval is, it suggests that reform is needed to strike a balance between rewarding scientific research and keeping prices down to Earth.
Moreover, Marathon could sell the review voucher for hundreds of millions of dollars.
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