Edaravone, marketed as Radicava by Mitsubishi Tanabe Pharma in the US, is a nootropic and neuroprotective agent that acts to scavenge free radicals, protecting against oxidative stress, which is a probable factor in ALS onset and progression. In 2015 it was approved for treatment of ALS in Japan after a four year clinical trial.
Eric Bastings, MD, of the neurology products division in the agency’s Center for Drug Evaluation and Research, said:
“This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option.”
Edaravone And Amyotrophic Lateral Sclerosis
It has been estimated that 5,000-6,000 people in the United States are diagnosed each year with amyotrophic lateral sclerosis. The disease is incurable, but edaravone has been shown to to slow down functional decline in patients by about one third.
“This is an important time for people living with ALS. The approval of Radaviva gives great promise for what we hope will be the first of many new treatments. We applaud the work MT Pharma America and the FDA are doing as they have taken unprecedented steps to get this treatment into the hands of patients as quickly as possible.”
The cost for edaravone is set at $1,086 per treatment, Mitsubishi Tanabe Pharma said. As the drug is administered is cycles consisting of 10 doses over 14 days and no dose the next 14 days, the annual cost would be $145,524.
Efficacy of edaravone for the treatment of ALS was shown in a six-month clinical trial conducted in Japan. In the trial, 137 participants were randomized to receive edaravone or placebo. At Week 24, individuals receiving edaravone declined less on a clinical assessment of daily functioning compared to those receiving a placebo.
The only other drug approved for ALS treatment is Sanofi Pharmaceuticals’ Riluzole, which can increase survival by two or three months, but at almost US $10 per tablet it is prohibitively expensive given the modest benefit to patients.
ALS, sometimes called Lou Gehrig’s disease, attacks the nerve cells in the brain and the spinal cord responsible for controlling voluntary muscles, such as those needed to move, speak, eat and breathe. It is one of the most well-known neuromuscular diseases, affecting approximately two in 100,000 people worldwide.4,5 While it is inherited in 5%–10% of cases, the cause for the majority of cases is not well understood but may involve genetic and environmental factors.
Image: James N. Sleigh, Wellcome Images. Motor nerves (green) found in the spinal cord project out long, thin axons to the periphery, where they contact muscles (black background), forming specialised connections or synapses called neuromuscular junctions (NMJs, red).