Catalyst Pharmaceuticals Confirms Sufficient Supply of Firdapse

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by Mary Chapman |

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Catalyst Pharmaceuticals update

In a business update amid the COVID-19 pandemic, Catalyst Pharmaceuticals said it has ample supplies of Firdapse (amifampridine), its treatment for Lambert-Eaton myasthenic syndrome (LEMS).

Besides having no known disruptions to Firdapse production, the company assured its treatment inventories will last at least through June 2021. Catalyst’s U.S. manufacturing partners also have implemented contingency plans to enable operations throughout the viral outbreak.

Catalyst recently said patients who need early medicine refills or increased-volume shipments during the outbreak should contact the program Catalyst Pathways.

“[W]ell before the COVID-19 pandemic, we had worked diligently to establish our supply chain to ensure that LEMS patients would have access to Firdapse without interruption, no matter the circumstances,” said Patrick J. McEnany, Catalyst’s chairman and CEO, in a press release.

“We are committed to protecting the safety and well-being of our personnel, the entire LEMS community, healthcare providers, patients in clinical trials, and clinical trial site employees, and this will remain our priority throughout these challenging times,” McEnany said.

LEMS is caused by autoantibodies — immune system proteins that wrongly target the body’s own tissues — specifically attacking voltage-gated calcium channels (VGCC) at nerve endings. Normally, these channels enable nerve cells to take up calcium, which is needed for the release of tiny vesicles containing the neurotransmitter acetylcholine. This chemical messenger then prompts muscle contraction and nerve-muscle communication.

Firdapse is a small molecule intended to block a channel on nerve cells — known as voltage-dependent potassium channel. This causes the opening of existing healthy VGCCs, allowing calcium ions to flow into neurons, restoring the release of acetylcholine into the neuromuscular junction, and improving muscle function.

Originally developed by BioMarin,  which markets the therapy in Europe, Firdapse was approved in 2018 by the U.S. Food and Drug Administration to treat LEMS symptoms in patients 17 and older. The European Medicines Agency had approved the therapy in 2009.

In response to the pandemic, Catalyst also said it is supporting the First Responders Children’s Foundation’s COVID-19 Emergency Response Fund, which was created to provide emergency grants to first responders’ families. For the past 20 years, the foundation has been providing college scholarships to children of first responders who have lost a parent in the line of duty.

“The support of Catalyst has allowed the foundation to provide much-needed financial assistance to first responders on the front lines during these challenging times,” said Jillian Crane, the foundation’s president.

Catalyst said it would announce its first-quarter net revenue results next month and that will continue to assess the pandemic’s financial impact, noting that it has been minimal to date.

To minimize risk due to the new coronavirus, the company implemented measures such as a travel ban and a work-from-home policy for all employees.

Among its clinical development programs, the company is evaluating Firdapse as a treatment for patients with anti-MuSK antibody positive myasthenia gravis. A Phase 3 clinical trial (NCT03304054) is nearly finished, but the company says that worldwide travel restrictions and social distancing requirements have delayed activities necessary to conclude the study.