ALS survival benefits with Radicava to be studied as part of collaboration
Treatment is approved in U.S., Canada, but not in Europe
Mitsubishi Tanabe Pharma Canada (MTP-CA) is teaming up with the Canadian Neuromuscular Disease Registry (CNDR) in Canada’s first real-world, evidence-generating study of Radicava (edaravone) in amyotrophic lateral sclerosis (ALS).
Data are expected to provide real-life insights into the survival benefits of Radicava in the progressive neurodegenerative disorder, thought to affect about 3,000 Canadians.
When Radicava was approved in Canada in 2018, it became the first new ALS treatment in that country in nearly two decades. The treatment is infused into the bloodstream and an oral formulation called Radicava Oral Suspension was approved Canada last year. The effect of edaravone — the active ingredient in both formulations — on real-world survival in ALS hasn’t been established, however.
“Edaravone has previously been shown to slow disease progression in people with ALS over six months; however, further real-world effectiveness evidence is needed,” Agessandro Abrahao, MD, CNDR’s co-principal investigator, said in a press release.
What does the real-world Radicava study seek to accomplish?
Edaravone is known as a free radical scavenger, meaning it extracts the reactive molecules that contribute to oxidative stress, a type of cellular damage implicated in ALS. Neurons are especially sensitive to oxidative stress. By removing them, the therapy should mitigate nerve damage and slow disease progression.
While Radicava is approved in Canada, the U.S., and some other countries, the evidence showing its effectiveness isn’t settled and regulators in Europe and other regions have declined to approve it.
“The objective of this study is to describe real-world survival effectiveness over a longer time frame,” said Abrahao, a neurologist and associate scientist with the Sunnybrook Health Sciences Centre in Toronto who is co-leading the study.
“This collaboration with the CNDR is part of our legacy in Canada and ongoing commitment to benefit patients, caregivers, and loved ones touched by rare diseases, including ALS,” said Andy Zylak, president of Mitsubishi Tanabe Pharma Canada.
The study is enrolling ALS patients across Canada whose symptoms started in the past three years. So far, about 182 patients on Radicava for at least six months between 2017-2022 and 860 controls on other medications have signed on, Abrahao announced in a poster presentation in June at the 2023 Canadian Neurological Sciences Federation Congress.
Its main goal is to see if Radicava can extend the time until a tracheostomy, or a tube in the neck to help with breathing, is needed. This outcome is known as tracheostomy-free survival.
“Real-world data provides additional insight into treatment for rare diseases like ALS, which can sometimes be challenging to research and evaluate in clinical trials,” said Dung Pham, medical affairs director, Mitsubishi Tanabe Pharma Canada. “Through our collaboration with the CNDR, we hope to gain important real-life insights into the role of Radicava in the treatment of ALS — insights that will ultimately benefit people living with this progressive, life-threatening disease and their families.”
“Access to innovative therapies is an urgent issue for people living with ALS,” said Tammy Moore, president of ALS Canada. “Understanding how innovative treatments perform in a real-world setting can assist stakeholders from across the healthcare system with decision making and potentially lead to faster access for people living with ALS and improved health outcomes.”
The Canadian Neuromuscular Disease Registry is an independent registry of people — more than 4,500 to date — with a neuromuscular disease. It works to improve understanding of such disorders and accelerate the development of treatments.