FDA Extends Catalyst’s Firdapse Approval to Patients, 6 and Older

The move followed the invalidation of Ruzurgi's approval by the agency

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has expanded its approval of Firdapse (amifampridine) to cover pediatric patients, its maker Catalyst Pharmaceuticals announced.

This means the therapy is approved in the U.S. to treat people with Lambert-Eaton myasthenic syndrome (LEMS) ages 6 and older.

“We are very pleased to have received FDA approval for the expanded pediatric indication for Firdapse. While the U.S. LEMS pediatric population is an exceptionally small number of patients, this positive outcome helps ensure that all eligible LEMS patients have access to Firdapse for the treatment of this rare disease,” Patrick McEnany, chairman and CEO of Catalyst, said in a press release.

The company offers a program called Catalyst Pathways to help eligible patients access Firdapse. Additional information about the program can be found online or by calling 1-833-422-8259.

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The approval of Firdapse for children with LEMS came just a few months after the FDA had invalidated its approval of Ruzurgi, a therapy developed by Jacobus Pharmaceutical that had been previously approved to treat children with LEMS in the U.S.

Ruzurgi contains the same active ingredient as Firdapse, a molecule called amifampridine, that improves muscle strength by boosting the power of the chemical signals sent from nerve cells to muscles, instructing them to contract.

After Ruzurgi was approved in 2019, Catalyst sued the FDA, alleging the agency’s decision to approve a therapy containing the same active ingredient infringed on the marketing exclusivity rights the FDA had awarded Catalyst when Firdapse was still an experimental medication and received its orphan drug designation.

A federal appeals court sided with Catalyst in January, leading the FDA to invalidate Ruzurgi’s approval, and  Catalyst soon after announced plans to seek approval of Firdapse for children with LEMS.

The FDA’s decision to extend Firdapse for pediatric LEMS patients “represents our long-standing and unwavering commitment to the LEMS patient community, and we are pleased that this medicine is now available for this important patient population,” McEnany said. “We thank the Agency for their collaboration during the review of this application and our Catalyst employees for their shared commitment to improving the lives of patients.”

Catalyst acquired the rights to Ruzurgi in a settlement earlier this year, bringing the three-year legal battle with its maker, Jacobus Pharmaceutical to an end.