US Court Won’t Rehear Its Ruling Overturning Ruzurgi’s FDA Approval

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by Marisa Wexler MS |

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A federal appeals court has denied Jacobus Pharmaceutical’s petition to rehear its prior ruling overturning the U.S. approval of the company’s Ruzurgi to treat children with Lambert-Eaton myasthenic syndrome (LEMS).

The decision is a victory for Catalyst Pharmaceuticals, which sued the U.S. Food and Drug Administration over Ruzurgi’s approval in 2019.

The issue at hand is that the active agent in Ruzurgi, called amifampridine, is also the active ingredient in Catalyst’s Firdapse, which was granted the FDA’s orphan drug designation and approved in 2018 to treat LEMS patients 17 and older.

The designation is given to promote the development of treatments that can improve care for rare conditions, and it comes with certain benefits, including a seven-year period of marketing exclusivity upon approval.

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How Will an Appeals Court Ruling on Ruzurgi Affect My Daughter?

In its suit, Catalyst alleged that the FDA had violated the company’s right to market exclusivity for amifampridine to treat LEMS when the agency approved Ruzurgi to treat patients ages 6–16 in 2019. A District Court upheld the FDA’s approval, but it was overturned by the 11th Circuit Court of Appeals, which has now denied Jacobus’s petition to rehear the case.

Catalyst is offering a program called Catalyst Pathways to help adults on Ruzurgi switch to Firdapse. While the program is not designed for children, Catalyst has stated that its staff are available to help parents of children with LEMS find ways to get access to Firdapse, such as via an expanded access program.

“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,” Patrick J. McEnany, chairman and CEO of Catalyst, said in a press release.

“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,” McEnany added.