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An unusual, decade-long fight over an orphan drug market appears to be at an end after a small, family-run company was defeated in a bruising battle with a rival and has now lost regulatory approval of its medicine.

Late last month, a federal appeals court refused to revisit a ruling that the U.S. Food and Drug Administration improperly approved a drug used to treat a rare neuromuscular disorder called Lambert-Eaton myasthenic syndrome, or LEMS, which is sold by Jacobus Pharmaceuticals. As a result, the FDA last week withdrew final approval of the medicine. Shipments to patients are already winding down.


The decision handed a victory to Catalyst Pharmaceuticals (CPRX), a publicly traded company that waged a bitter fight with Jacobus over a market numbering no more than a few thousand patients in the U.S. and, in the process, became a symbol of the growing tensions over targeting small patient populations with high-priced treatments.

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