FDA approves Marathon Pharmaceuticals' steroid drug costing $89k, raising question about orphan drug status

The FDA approved Northbrook, Ill.-based Marathon Pharmaceuticals' drug, deflazacort, which treats Duchenne muscular dystrophy, according to the Washington Post.

Here are five things to know:

1. The drug has an $89,000 annual list price. The drug's net price will total $54,000 annually after accounting for rebates and discounts, according to the company's CFO Babar Ghias.

2. To obtain approval, Marathon Pharmaceuticals went through the orphan drug approval process. The FDA implemented this process for companies developing drugs that treat very rare diseases.   

3. The company can sell the drug in the United States exclusively for seven years with the orphan designation. While the drug recently received U.S. approval, deflazacort was available in other countries.

4. Obtaining approval for existing drugs is not a new trend as companies have made major profits from this practice, the Post reports. In 2008, the FDA approved an orphan drug to treat Huntington's disease that was previously available abroad. The drug had a $42.28 list price in 1998 for a bottle at a European pharmacy. However, this price soared to more than $6,000 in the United States after receiving orphan drug status a decade later.

5. The FDA also granted Marathon Pharmaceuticals a "priority review" voucher, which means the agency will review the drug at a quicker rate or Marathon can sell the drug to another company, according to the Post. While the vouchers have good intentions in motivating companies to invest in developing drugs that treat rare diseases, some medical industry experts question if companies are not applying the vouchers correctly.

Aaron Kesselheim, MD, JD, an associate professor of medicine at Boston-based Harvard Medical School, told the Post, "How many examples of this do we have to see before we can start to rethink the priority review voucher as a means of incentivizing innovation? This also seems to be another example of gaming the Orphan Drug Act, which was intended to try and encourage research into new therapeutic entities for people who have rare diseases — and it doesn't seem like this is that."

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