The first new FDA approval for ALS in 22 years, here's what you should know.
The FDA approved edaravone (Radicava, Mitsubishi Tanabe Pharma America) on Friday, May 5, 2017 for the treatment of amytrophic lateral sclerosis (ALS) also known as Lou Gehrig's Disease.1 The approval is only the second medication approved for the indication, with riluzole (Rilutek) being approved in 1995. Radicava has been available in Japan and Korea since 2015.1
The approval is based on a 24-week, phase 3 double-blind, parallel-group study comparing edaravone to placebo. The primary outcome was functioning as defined by ALS Functional Rating Scale-Revised (ALSFRS-R).2 The ALSFRS-R evaluates functional abilities such as swallowing and speech, and higher scores are associated with higher functioning. The trial results showed ALS patients treated with edaravone had less functional loss at 6 months compared to the placebo group with a between group difference of 2.49 +/- 0.76 (P = .001). It is important to note none of the trials for edaravone in ALS have yet to evaluate the effect on survival.
Radicava is an intravenous treatment given as 60 mg daily for a 14-day cycle followed by a 14-day drug-free period. Subsequent cycles administer Radicava daily for 10 days over a 14-day period followed by a 14-day drug-free period again.3*The mechanism of edaravone in ALS is not well understood but hypothesized to be related to oxidative stress on vascular endothelial cells and nerve cells. Renal function and hepatic function should be closely monitored while patients receive treatment with edaravone. The most common adverse events are bruising and gait disturbance.
Rare but significant adverse events include hives, swelling, anaphylaxis due to the inclusion of sodium bisulfite in patients with a sulfite allergy.
1. FDA approves drug to treat ALS [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm557102.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed May 8, 2017.
2. Tanaka M, et al. A 24-week, phase III, double-blind, parallel-group study of edaravone (MCI-186) for treatment of Amytrophic Lateral Sclerosis. Neurology. 2016; (86):16. SP3 189.
3. Radicut(R) [Package Insert]. Osaka, Japan. Mitsubishi Tanabe Pharma Corporation; June 2015.