Judgment in Favor of Catalyst Vacates FDA Approval of Ruzurgi

A federal appeals court in the U.S. has issued a mandate that effectively vacates the 2019 regulatory approval of Jacobus Pharmaceutical’s Ruzurgi for the treatment of children with Lambert-Eaton myasthenic syndrome (LEMS).

The ruling, by the U.S. Court of Appeals for the 11th Circuit, directs a lower district court to enter a win — called a summary judgment — in favor of Catalyst Pharmaceuticals in its overarching lawsuit against the U.S. Food and Drug Administration (FDA), the company said in a press release.

Catalyst had sued the FDA in 2019 over the agency’s approval of Ruzurgi for LEMS patients, ages 6–16, on the grounds that the federal regulator had violated the company’s right to market exclusivity for its own medication, Firdapse (amifampridine), for treating LEMS.

Amifampridine, the active ingredient of Firdapse, is also the main component used in Ruzurgi.

Firdapse, prescribed to patients ages 17 and older with LEMS, was initially granted the FDA’s orphan drug designation in 2018 — and then final approval that same year.

The issue is that the FDA’s orphan drug status, which aims to advance the development of therapies that can improve care for rare disorders, also grants seven years of marketing exclusivity to developers if and when their treatment is approved. In its suit, Catalyst had claimed that the FDA’s approval of Ruzurgi was a violation of Firdapse’s market exclusivity.

In January, a federal appeals court rejected Jacobus’ petition to rehear its previous ruling that overturned federal approval of Ruzurgi. The FDA’s approval of Ruzurgi was initially upheld by a district court, but that ruling was overturned by the 11th Circuit, based in Atlanta, which ultimately turned back Jacobus’ petition.

Catalyst currently has in place a program designed to help adults being treated with Ruzurgi transition to Firdapse. The program, called Pathways, is not meant for children, but Catalyst has said it can help parents of children who have LEMS identify means to gain access to Firdapse, such as through an expanded access program.

“Catalyst’s priority has always been, and will continue to be, to put patients’ needs first, and we are well prepared to address their questions and do everything we can to ensure that all LEMS patients continue with uninterrupted access to amifampridine for treating their LEMS condition, whether through commercial access or compassionate use access for those who qualify,” said Patrick J. McEnany, Catalyst’s chairman and CEO.

“Our patient-focused Catalyst Pathways team stands ready to provide information to patients currently being treated with Ruzurgi on how best to transition to Firdapse,” McEnany added.

Information for prescribers and patients regarding the Pathways program is available here and by calling 833-422-8259.