Catalyst, DyDo Strike Deal to Develop, Market Firdapse in Japan

Catalyst Pharmaceuticals has teamed up with DyDo Pharma to develop and commercialize Firdapse (amifampridine) in Japan for the treatment of Lambert-Eaton myasthenic syndrome (LEMS).

Under the terms of their agreement, DyDo will be responsible for funding all clinical, regulatory, marketing, and commercialization activities in Japan. In turn, Catalyst will be responsible for supplying the oral small molecule medication, as well as supporting Dydo in its efforts to seek Firdapse’s approval in the country.

“DyDo Pharma’s experienced team and commitment to rare diseases makes the company an attractive choice for Catalyst for the development and distribution of our amifampridine product for the treatment of LEMS in Japan,” Patrick J. McEnany, chairman and CEO of Catalyst, said in a press release.

“Given the limited availability of treatment options for LEMS in Japan, we look forward to working with DyDo Pharma to address this unmet medical need by hopefully providing a new treatment option for patients and their families,” McEnany said.

LEMS is caused by the body’s immune system wrongly launching an attack against calcium channels found in nerve cell endings. As a result, the nerve cells become less able to release acetylcholine, a signaling molecule that plays a key role in muscle contraction, ultimately leading to muscle weakness and other symptoms of LEMS.

Firdapse is an oral medication that works by increasing the release of acetylcholine from nerve cells, potentially improving patients’ muscle function. The medication is available in 10 mg tablets, and doses are tailored to suit each individual’s needs.

In clinical trials, treatment with Firdapse has been shown to improve muscle strength and various measures of physical function in people with LEMS. As such, the therapy was found to improve patients’ quality of life.

In the U.S., Firdapse is approved to treat LEMS patients ages 17 and older. It also is approved for the same indication in the European Union and in Canada.

In Japan, Firdapse has been granted orphan drug designation by the Japanese Ministry of Health, Labour and Welfare. This designation is given to therapies that have the potential to treat rare diseases, defined as those affecting fewer than 50,000 people in Japan, and for which there is a high medical need. Such status also gives the therapy’s developer access to certain incentives, like subsidies and faster regulatory review.

“We are very pleased to have acquired a license for the Japanese rights for Firdapse, which Catalyst sells in the United States,” said Yasunori Inaoka, president and representative director of DyDo.

“Going forward, we will pursue development with the goal of obtaining marketing authorization approval for Firdapse as a treatment for LEMS in Japan, so that we can offer a new therapeutic option to Japanese LEMS patients,” Inaoka said.