Edaravone (marketed as Radicava®) is a free radical scavenger that was approved for the treatment of amyotrophic lateral sclerosis (ALS) in Japan (2015), South Korea (2018), and the United States (2017). The pivotal trials demonstrated that edaravone can slow functional decline in a subset of ALS patients with early disease and relatively rapid progression[1].
Edaravone represents one of only two FDA-approved disease-modifying therapies for ALS, alongside riluzole, making it a critical treatment option for patients diagnosed with this progressive neurodegenerative disease.
| Parameter | Value |
|---|---|
| Phase | Phase 3 |
| Status | Approved (2017) |
| Drug | Edaravone (Radicava®) |
| Dosage | 60 mg IV infusion daily |
| Patient Population | Adults with ALS |
| Treatment Cycle | 28 days on, 28 days off |
| ClinicalTrials.gov Identifier | NCT01492686 |
| Sponsor | Mitsubishi Tanabe Pharma |
Edaravone works through multiple neuroprotective pathways that address the key pathological mechanisms underlying ALS[2]:
The drug's ability to cross the blood-brain barrier allows it to directly protect motor neurons in the central nervous system. Research suggests edaravone may work through:
The clinical development program included multiple randomized, double-blind, placebo-controlled trials:
A critical post-hoc analysis identified a subgroup of patients who showed the strongest response to edaravone:
This analysis formed the basis for the FDA's indication, which specifies treatment for "patients with ALS."
| Patient Group | Treatment Effect |
|---|---|
| All patients | Modest benefit (not significant) |
| Early disease, fast progression | Significant benefit (p=0.013) |
| Later disease | No significant benefit |
The FDA-approved dosing schedule:
Clinical practice guidelines recommend edaravone for:
Edaravone established several important precedents[3]:
As of 2024, edaravone is used in clinical practice alongside:
Long-term studies continue to evaluate[4]:
Edaravone ALS Study Group. Efficacy and safety of edaravone for amyotrophic lateral sclerosis. The New England Journal of Medicine. 2017. ↩︎ ↩︎
Jia J, et al. Mechanisms of neuroprotection by edaravone in amyotrophic lateral sclerosis. Neuropharmacology. 2019. ↩︎
Yoshino H. Edaravone for the treatment of amyotrophic lateral sclerosis. Expert Opinion on Pharmacotherapy. 2018. ↩︎
Takahashi K, et al. Long-term safety and efficacy of edaravone in ALS. Journal of the Neurological Sciences. 2021. ↩︎