Catalyst Expects No Supply Problems for Firdapse Due to COVID-19

Catalyst Pharmaceuticals says the COVID-19 pandemic is not likely to affect North American supplies of Firdapse (amifampridine), its Lambert-Eaton myasthenic syndrome (LEMS) treatment.

“We assure the LEMS community that we have product available and anticipate no disruptions to our manufacturing facilities, as they are all in the U.S. and Canada,” the company said on its website.

Patients who wish to have additional medicine on hand during the outbreak should contact Catalyst Pathways. The free personalized program, which offers patients and families support throughout their treatment journey, can work with insurers to potentially provide access to early Firdapse refills or increased-volume shipments. Visit the program website or call 833-422-8259 for more information.

LEMS is an autoimmune disease that affects the neuromuscular junction, the area where nerve cells and muscle fibers communicate.

LEMS is caused by the production of autoantibodies — antibodies that wrongly target the body’s own tissues — specifically targeting voltage-gated calcium channels (VGCC) located at nerve endings. Normally, those channels enable nerve cells to take up calcium, necessary for the release of tiny vesicles containing the neurotransmitter acetylcholine, a chemical messenger that prompts muscle contraction and nerve-muscle communication.

Firdapse is a small molecule intended to block another channel on nerve cells — called the voltage-dependent potassium channel. This results in the opening of any residual healthy VGCCs, permitting calcium ions to stream into nerve cells, restoring the release of acetylcholine into the neuromuscular junction, and enhancing muscle function.

Originally developed by BioMarin and marketed by that company in Europe, Firdapse was approved in 2018 by the U.S. Food and Drug Administration to treat symptoms of LEMS in patients age 17 and older. The European Medicines Agency approved the therapy in 2009. Last year, Catalyst expanded its license with BioMarin to potentially market Firdapse in Japan.

The company now aims to broaden Firdapse’s use to other diseases, including myasthenia gravis and spinal muscular atrophy type 3, and congenital myasthenic syndromes.

Catalyst recently announced it expected to report 2019 net product revenues of $102 million for Firdapse. In addition, it said it anticipates net product revenues this year of $135 million to $155 million, and that it plans to introduce unspecified programs in 2020 to assist people with LEMS and their healthcare providers.