Court Supports Catalyst’s Appeal for Market Exclusivity for Firdapse

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A federal appeals court has ruled that the U.S. Food and Drug Administration (FDA) should not have approved Ruzurgi (amifampridine) for the treatment of children with Lambert-Eaton myasthenic syndrome (LEMS).

The decision by the U.S. 11th Circuit Court of Appeals is a win for Catalyst Pharmaceuticals, which sued the FDA and several other parties over Ruzurgi’s approval in 2019, stating it violated the company’s right to orphan drug exclusivity for Firdapse — a therapy chemically similar to Ruzurgi and approved for adults with LEMS. An earlier District Court decision had upheld the FDA’s approval of Ruzurgi.

“We are extremely pleased with the 11th Circuit Court’s decision,” Patrick J. McEnany, CEO and chairman of Catalyst, said in a press release.

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When Catalyst was developing Firdapse, the FDA granted it an orphan drug designation. The designation is given to experimental treatments with the potential to substantially improve care for patients with rare diseases. It confers certain incentives to a therapy’s developer, including seven years of market exclusivity if the therapy ultimately is approved.

“The purpose of the Orphan Drug Act is to encourage pharmaceutical companies to make the R&D [research and development] investments necessary to bring FDA-approved therapies to patient populations living with very rare diseases, and the biggest incentive the Orphan Drug Act provides is the ability to have an exclusive market within that rare disease for their drug for seven years once they obtain approval,” McEnany said.

The act has been “proven successful for the more than 350 rare diseases that now have an FDA-approved treatment … and the incentives to develop drugs to treat these rare diseases need to remain intact,” he added.

In striking down the earlier District Court decision, the appeals court essentially agreed with Catalyst’s argument that the approval of Jacobus Pharmaceutical’s Ruzurgi violated Catalyst’s right to market exclusivity for Firdapse.

The higher court has now remanded the matter to the District Court with instructions to enter summary judgment for Catalyst.

“This decision increases the hope for the patients living with these diseases that one day soon an approved drug for their rare disease might be a reality,” McEnany said. “This is a goal that we feel we share with the FDA despite this dispute, and we look forward to working with the FDA in the future on these issues.”

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McEnany acknowledged that the ruling is likely to spark questions and concerns among LEMS patients who are currently on Ruzurgi, and their healthcare providers.

“Our priority has always been, and will continue to be, to treat patients needs first, and so we are prepared to address their questions and do everything we can to ensure that ALL patients will have uninterrupted access to amifampridine for treating their LEMS condition,” McEnany said.

The company offers a program called Catalyst Pathways that can help adults on Ruzurgi switch to Firdapse. While the program is not designed for children, its staff are available to help pediatric LEMS patients on Ruzurgi find ways to get access to Firdapse, such as through Catalyst’s expanded access program for the therapy.

Catalyst is also planning to discuss with the FDA the possibility of expanding Firdapse’s approval to cover children, though the company noted that this is not guaranteed and will take time.