Skip to Main Content

The next chapter in an unusual saga involving a high-priced, rare disease drug will play out in a federal court, where Catalyst Pharmaceuticals (CPRX) has accused the Food and Drug Administration of violating the law when it recently approved a similar medicine made by a small, family-run company. And the battle is being closely watched for clues that incentives for developing such drugs may be jeopardized.

In its lawsuit, Catalyst alleges the FDA “arbitrarily and capriciously” approved the other medicine, which is made by Jacobus Pharmaceutical, by applying different standards. Moreover, the agency unfairly trampled on Catalyst’s right to seven years of exclusive marketing, a designation for so-called orphan drugs that treat small numbers of patients, according to the lawsuit.


In doing so, the FDA improperly waded into a heated debate over pricing by unfairly opening the door to off-label marketing of the Jacobus drug, the lawsuit stated. The Catalyst drug was approved to treat adults with Lambert-Eaton myasthenic syndrome, or LEMS, a neuromuscular disorder that afflicts a few hundred to a few thousand people in the U.S. The Jacobus drug is approved only for treating youngsters.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!