Health Canada has accepted Mitsubishi Tanabe Pharma Corporation’s New Drug Submission (NDS) for edaravone to treat patients with amyotrophic lateral sclerosis (ALS). Health Canada has also granted the drug a Priority Review so that it should take less than 6 months for the review process to be completed.
If approved by Canada, the drug will still have to be reviewed by other agencies such as Canadian Agency for Drugs and Technologies in Health (CADTH) and later the Provincial Ministries. For all of those reviews, it is important to not only have the manufacturer of the drug involved but also the patients. To that end, ALS Canada is in contact with those agencies to make sure the patients’ voices are also heard.
Recently, we talked with Tammy Moore, executive director of ALS Canada about the NDS filing and she said her group is in contact with all parties to make sure patients are involved in the review process. And they are talking not just with Health Canada but with the other agencies to help streamline the process so that patients can gain access to the drug in a timely manner. Right now, the Priority Review means the drug may get approved in 6 months but it can take many more months, if not years, for CADTH and the Provinces to complete their review process.
ALS is a neurodegenerative disease that afflicts about 3000 Canadians, with the majority of them dying within 2 to 5 years of diagnosis but some living for several decades (e.g., Steven Hawking). Symptoms begin gradually and can often go unnoticed (clumsy behavior, shortness of breath, decreased muscle tone) but as the disease progresses, persons will lose the ability to move, talk, swallow, and breath. At present, there is no cure or treatment that can attenuate the progression of the disease but that may change if edaravone gets approved.
In 2015, the drug was first approved in Japan, where the phase 3 clinical trial was conducted. In that study, 137 ALS patients received edaravone or placebo and the study found that edaravone attenuated patients’ daily functioning compared to placebo. More specifically, the group receiving edaravone had a Revised ALS Functional Rating Scale (ALSFRS-R) score of -5.01 compare to a score of -7.50 for the group receiving the placebo.
The most common adverse reactions with edaravone were bruising (contusion) and gait disturbance.
In May 2017, Radicava (edaravone) was approved by the U.S. Food and Drug Administration (FDA). That approval was largely based on that phase 3 trial in Japan. To date, no advanced clinical trials with the drug have been completed in North America.
The FDA also noted that edaravone may cause hives, swelling, or shortness of breath, and allergic reactions to sodium bisulfite, an ingredient in the drug.
Edaravone is an antioxidant and while it is not clear how it works in relieving ALS symptoms, it is hypothesized that oxidative stress is involved in the pathophysiology of ALS.
For more information about ALS, visit www.als.ca