Canada Accepts New Drug Submission, Grants Priority Review for Edaravone to Treat ALS

Health Canada accepted Mitsubishi Tanabe Pharma’s new drug submission (NDS) for edaravone as an intravenous treatment option for amyotrophic lateral sclerosis (ALS).

Edaravone is currently approved as a treatment for ALS in Japan, South Korea, and the United States (where it is marketed by Mitsubishi Tanabe as Radicava).

Health Canada also granted priority review to the NDS, according to Mitsubishi Tanabe and Mitsubishi Tanabe Pharma America (MTPA).

A distribution protocol was established in March to prepare the infrastructure and business operations necessary to bring edaravone to Canadian patients if it is approved by Health Canada so it will be available to Canadian patients as soon as possible.

The NDS was filed on March 9. New drug submissions granted priority review are subject to a regulatory review target of 180 days from the date Health Canada accepts the filing.

“We are dedicated to delivering innovative products that address the unmet medical needs of patients facing serious and life-threatening diseases,” Atshushi Fujimoto, president of MTPA, said in a press release. “The clinical development effort for edaravone in ALS spanned 13 years and included the pivotal Phase 3 study MCI186-19, which forms the basis for the company’s NDS submission.”

Radicava (edaravone) was the first treatment approved specifically for ALS by the U.S. Food and Drug Administration in more than two decades.

Edaravone slows the decline of physical functioning in ALS patients by removing free radicals in the nervous system. Free radicals are a normal part of the cellular process of producing energy and are supposed to be removed by the body quickly after serving their role.

But when they are not eliminated, they can cause oxidative stress, which is thought to lead to damage and cell death in ALS.

Both FDA approval and Health Canada’s NDS submission were based on the pivotal Phase 3 MCI186-19 clinical trial (NCT01492686), which evaluated the effectiveness and safety of ederavone in treating ALS.

Now completed, the primary objective was to confirm the efficacy of 60 mg of edaravone via intravenous drip infusion once daily in patients with ALS, based on the changes in the revised ALS functional rating scale (ALSFRS-R) scores after 24 weeks.

Patients were divided into two groups: one receiving 60 mg of edaravone intravenously and another receiving placebo. Although progression varies from person to person, research has shown that people with ALS lose an average of one ALSFRS-R point per month.

After six months of treatment (24 weeks), those who received edaravone experienced a decline that was 33 percent less in the ALSFRS-R score compared to those receiving placebo, which was considered a significant achievement.

In results published in the journal The Lancet Neurology, researchers concluded that edaravone could effectively reduce the progression of disability in ALS.