$375K rare disease drug gets unforeseen competition after FDA approval

The FDA has approved a new medication for a rare neuromuscular disorder in children, adding unforeseen competition to Catalyst's $375,000 drug, Firdapse, which was at the center of a pricing controversy earlier this year, according to STAT news.

Jacobus Pharmaceutical received FDA approval May 6 for its rare disease drug for Lambert- Eaton myasthenic syndrome for children ages 6 to 17. The disorder affects the connection between nerves and muscles, causing weakness in affected patients.

The approval of Jacobus' Ruzurgi adds potentially unforeseen competition to Firdapse, which was approved for LEMS in adults.

Although the two drugs are for different age ranges, providers may be able to prescribe Ruzurgi as an off-label drug to treat adult patients, competing with Firdapse.

The CEO of Jacobus, Laura Jacobus, told STAT her company will price Ruzurgi lower than Firdapse. Catalyst shares plummeted nearly 40 percent after the news.

The controversy surrounding Firdapse, a formerly inexpensive drug, began in December when it hiked the price of the drug to $375,000 a year after the FDA approved it. Patients had formerly received the drug, used off-label to treat the rare disease, for free from Jacobus Pharmaceuticals..

In February Sen. Bernie Sanders, I-Vt., called the pricing decision "a blatant fleecing of American taxpayers" and an "immoral exploitation of patients."

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