Catalyst Plans to Request FDA Extend Use of Firdapse to Children

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Catalyst Pharmaceuticals is planning to request that the approved use of Firdapse in the U.S. be expanded to include children with Lambert-Eaton myasthenic syndrome (LEMS).

The company also announced, as part of its “patient first” commitment, an intention to sustain established access programs so that anyone with LEMS seeking treatment with Firdapse can acquire the medication.

“As we enter 2022, we believe that our strong financial position will further empower us to execute on our strategic objectives,” Patrick J. McEnany, chairman and CEO of Catalyst, said in a press release.

The U.S. Food and Drug Administration (FDA) recently invalidated its approval of Jacobus Pharmaceutical’s Ruzurgi to treat children with LEMS. The move came after Catalyst sued the FDA over Ruzurgi’s 2019 approval.

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Firdapse and Ruzurgi both contain the same active agent, a molecule called amifampridine. When Catalyst was developing Firdapse, the FDA named the then-experimental medication an orphan drug. Among the benefits of this designation, given to incentivize therapy development for rare diseases, is a seven-year period of market exclusivity upon approval.

Firdapse was approved to treat LEMS patients ages 17 and older in 2018. In its suit, Catalyst alleged that the FDA had violated its right to market exclusivity when it approved Ruzurgi for LEMS patients, ages 6–16, the following year. A federal appeals court ultimately sided with Catalyst.

“2021 was a landmark year for Catalyst as we achieved record revenues while attaining a significant milestone in upholding our Orphan Drug Exclusivity for Firdapse for the treatment of Lambert-Eaton myasthenic syndrome (LEMS). Our strong performance is representative of our exceptional … capabilities, as well as our continued commitment to the patients we serve,” McEnany said.

Catalyst offers a program called Pathways that can help adults on Ruzurgi transition to Firdapse. According to the company, although not specifically designed for children, the program may offer assistance to families of children who want to access Firdapse.

Catalyst plans to submit a pediatric supplemental new drug application to the FDA this year that might expand Firdapse’s approval to children with LEMS. The company also is planning to seek approvals or expanded approvals outside the U.S., starting in Japan and Canada. The oral treatment is approved for adults in Canada and Europe.

In addition, Catalyst intends to grow Firdapse’s use in the U.S. by raising awareness and educating patients and healthcare providers. A particular focus will be on patients who develop LEMS in association with cancer, a form of the condition sometimes referred to as paraneoplastic LEMS.

“As a result of our accomplishments, we expect to realize a meaningful increase in 2022 revenues and look forward to building upon this momentum to fully maximize the value of Firdapse,” McEnany said.