Phase 3 Trial Starts in Japan to Aid Firdapse’s Potential Approval

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by Steve Bryson, PhD |

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Catalyst Pharmaceuticals has announced its partner DyDo Pharma has launched a Phase 3 registrational trial in Japan to evaluate the safety and effectiveness of Firdapse (amifampridine) to treat Lambert-Eaton myasthenic syndrome (LEMS).

A registrational trial is designed to obtain sufficient clinical data to support the filing of an application for a therapy’s regulatory approval. Currently, Firdapse is not yet available to LEMS patients in Japan.

“We are pleased with the important progress being made by DyDo as we advance on our shared commitment of bringing a novel treatment option to LEMS patients in Japan,” Patrick McEnany, Catalyst’s chairman and CEO, said in a press release. “The initiation of this Phase 3 program marks an important milestone towards our goal to expand the global footprint of Firdapse for the treatment of LEMS.”

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In LEMS, the immune system mistakenly attacks calcium channels located in nerve cell endings. As a result, nerve cells release less acetylcholine — a signaling molecule that triggers muscle contractions. A lack of acetylcholine release leads to muscle weakness and other symptoms.

Firdapse is an oral medication — available in 10 mg tablets — that is designed to keep calcium channels opened for a longer period of time, thereby increasing the release of acetylcholine from nerve cells and improving muscle function.

It is approved to treat people with LEMS, 17 and older, in the U.S., Canada, and Europe. Clinical trials supporting its approval demonstrated Firdapse improved muscle strength, physical function, and quality of life in patients.

Recently, Catalyst entered into an agreement with DyDo to develop and commercialize Firdapse in Japan. Under the terms of the agreement, DyDo will fund the development and commercialization of the therapy in Japan, pending regulatory approval.

Catalyst’s role will be to supply Firdapse to DyDo for clinical and commercial purposes and provide technical support to obtain regulatory approval from Japanese authorities. Catalyst will also be entitled to milestones for development and sales and revenue for Firdapse’s clinical and commercial supply.

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Firdapse was named an orphan drug by the Japanese Ministry of Health, Labour, and Welfare. This designation is given to medications that have the potential to treat rare diseases, defined as those affecting fewer than 50,000 people in Japan. It also provides developers with financial subsidies and accelerated regulatory reviews.

“Currently, there are no approved treatments for this rare autoimmune neuromuscular disorder in Japan, and we believe Firdapse has the potential to provide a meaningful new therapy option to patients living with this disease,” McEnany said. “We appreciate the collaborative effort of our partnership and look forward to continuing to support DyDo in the clinical advancement of Firdapse.”