Radicava (edaravone, previously MCI-186) is the first treatment for amyotrophic lateral sclerosis (ALS) approved by the U.S. Food and Drug Administration (FDA) in more than 20 years.
How Radicava works
Radicava is a free radical scavenger. Free radicals can occur as a normal part of the cellular process of producing energy and are quickly removed by the body. However, if they remain, they can cause oxidative stress, leading to damage and cell death.
Oxidative stress caused by free radicals is believed to be one of the causes of nerve cell death in ALS. For example, in some familial ALS patients, an enzyme that removes free radicals, SOD1, is mutated and no longer works. Radicava has a neuroprotective effect and slows the progression of ALS by removing free radicals in the nervous system.
Radicava in clinical trials
FDA approval was based on the results of a double-blind, randomized, placebo-controlled Phase 3 study (NCT01492686), which evaluated the effectiveness and safety of Radicava in treating ALS. After a 12-week observation period, 137 participants were divided into two groups. Those in the first group received 60 mg of Radicava intravenously on a specified schedule, and participants in the second group received a placebo, both for six months.
The primary objective of the study was for the treatment group to improve their score in a measurement of disability progression known as the ALS Functional Rating Scale-Revised (ALSFRS-R). Although the rate of progression varies among patients, research has shown that people with ALS lose an average of one point per month on this scale.
After six months of treatment, those who received Radicava experienced a 33% lesser decline in the ALSFRS-R score compared with those in the placebo group. That figure was considered statistically significant, and researchers concluded that Radicava significantly reduces the progression of disability in ALS patients. The results of this study were published in the scientific journal The Lancet Neurology.
The most common adverse reactions from the treatment were walking problems, bruises, and headaches.
Radicava is administered intravenously (into the bloodstream) in 28-day cycles. The treatment starts with a patient receiving the therapy for 14 consecutive days, followed by a two-week drug-free period. Patients then receive an infusion during 10 of the next 14 days, followed by a two-week drug-free period.
Patients are prescribed 60 mg per treatment, and each treatment takes 60 minutes to administer.
It is important that patients make their healthcare provider aware of other medical conditions they have, which could lead to complications, including asthma and allergies to other medicines. They also should let their provider know if they are pregnant, or plan to become pregnant, or if they are breastfeeding or plan to breastfeed.
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