DyDo seeks approval of Firdapse for LEMS in Japan
Decision on whether to approve Firdapse expected in September
A decision from Japan’s Pharmaceuticals and Medical Devices Agency is expected in September.
DyDo’s application was submitted in collaboration with Catalyst Pharmaceuticals, which developed Firdapse. DyDo is leading the development of Firdapse in Japan under a 2021 agreement between the two companies.
“Given the absence of therapies available in Japan for this rare neuromuscular disorder, we believe that if approved, FIRDAPSE holds the potential to be a novel treatment option for Japanese individuals grappling with this condition,” Patrick McEnany, chairman and CEO of Catalyst, said in a company press release.
Catalyst to develop and market Firdapse in Asia, Central and South America
McEnany noted the submission triggers a payment to Catalyst, according to the 2021 deal.
It also triggers an expansion of Catalyst’s rights to develop and market the therapy in certain countries in Asia, as well as Central and South America, according to Catalyst, which didn’t specify the countries, stating only that it will focus on “the Asia Pacific and Latin American regions.”
“We look forward to initiating our plans to expand the reach for Firdapse into other global regions seeking a novel therapy for the treatment of LEMS,” McEnany said.
LEMS is an autoimmune disease caused by antibodies that target healthy nerve cells, disrupting the ability of these cells to send the signals to muscle cells that normally tell muscles to move. This ultimately results in symptoms such as muscle weakness. Firdapse is designed to strengthen the muscles in LEMS by basically boosting the power of signals from nerve cells.
Firdapse has been evaluated in two clinical trials (NCT01377922 and NCT02970162), which collectively enrolled more than 60 people with LEMS who were given the therapy or a placebo. Results from both studies showed, relative to the placebo, treatment with Firdapse led to less muscle weakness and better outcomes on reports of overall health measured by clinicians or by patients themselves.
Catalyst is currently seeking a change in Firdapse’s approval in the U.S., asking regulators to increase the maximum allowable daily dose of the therapy to 100 mg per day from 80 mg.