On Monday evening, the Food and Drug Administration found a way to dampen controversy over a $375,000 medicine. The agency approved a drug from Jacobus Pharmaceuticals, a small, family-run company, for treating a rare disease called Lambert-Eaton myasthenic syndrome, or LEMS, for children ages 6 to 17. The move potentially adds unforeseen competition for Catalyst Pharmaceuticals (CPRX), which only last December won an FDA endorsement to market its own treatment for adults.
The FDA approval of the Catalyst drug caused a stink. Until then, a few hundred LEMS patients were able for years to obtain the Jacobus drug for free under a compassionate use program sanctioned by the FDA. But with its approval, Catalyst obtained seven years of market exclusivity, which meant Jacobus and compound pharmacies could no longer market their versions. And while Catalyst runs patient assistance programs to offset the cost of its drug, taxpayers and insurers are footing the bill.
We spoke briefly with Laura Jacobus, who runs the small drug maker, and Gavin Heffernan, the director of chemistry, about their plans, although they declined to discuss the possibility that some physicians may prescribe their drug on an off-label basis for adults, which has Catalyst investors worried.