Firdapse approved to treat LEMS patients in Japan
Oral therapy, also available in US and Europe, works to improve muscle strength
Firdapse (amifampridine) has been approved in Japan to treat people with Lambert-Eaton myasthenic syndrome (LEMS).
The therapy will be supplied in the form of oral tablets containing 10 mg of amifampridine, according to Catalyst Pharmaceuticals, the company that holds Firdapse’s licensing rights in North America.
In collaboration with Catalyst, DyDo Pharma submitted an application requesting Firdapse’s approval to Japan’s regulatory health agency late last year. DyDo is responsible for the development of Firdapse in Japan following a 2021 agreement between the two companies.
Firdapse acts to increase the power of nerve cell signals to muscle cells
“We are pleased that our partner, DyDo, has secured regulatory approval for Firdapse in Japan. This represents a meaningful milestone, bringing renewed hope to patients and further affirming Firdapse’s proven effectiveness in the treatment of LEMS,” Richard J. Daly, Catalyst’s president and CEO, said in a company press release.
“We appreciate the collaborative efforts of our partner in securing this approval, and we remain focused on expanding our innovative rare disease product portfolio beyond the U.S. to make a meaningful impact on patients’ lives worldwide,” Daly added.
An autoimmune disease, LEMS is caused by the immune system wrongly launching an attack against calcium channels found in nerve cell endings, which are key to controlling muscle movement. Disease symptoms of muscle weakness, mostly in the lower limbs, result.
Firdapse is an oral treatment that’s designed to enhance muscle strength by increasing the power of signals sent from nerve cells to muscles that instruct them to contract, a process that’s impaired in LEMS.
Firdapse’s efficacy and safety were evaluated in two Phase 3 clinical trials — NCT01377922 and NCT02970162 — jointly involving more than 60 people with LEMS. Results showed that Firdapse was better than a placebo at easing muscle weakness. It also led to better outcomes on reports of overall health assessed either by clinicians or patients.
The oral therapy has been approved in the U.S. for adults and pediatric patients, ages 6 and older with LEMS, as well as in Europe and Canada for the treatment of adults with the disease.
Catalyst recently announced that the maximum allowed daily dose of Firdapse in the U.S. has been increased from 80 to 100 mg per day, which is expected to offer healthcare providers and patients more flexibility in treatment regimens.
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