The FDA has approved Radicava (edaravone) for the treatment of fatal neurological disease ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig’s disease. The drug is already marketed by Osaka-based Mitsubishi Tanabe in Japan and South Korea since 2015, and is expected to reach the US market by August.
The glutamate antagonist Rilutek (riluzole) was the first medication to be granted FDA approval for ALS, although the initial registration studies failed to meet the FDA’s typical approval standards. Instead of functional measures, which have been used historically, Rilutek’s approval was based on its survival benefits when compared with placebo, which was between three and six months. Since its market launch in 1996, Rilutek has remained the only approved therapy option for ALS patients in the US, but in any case it only has a very modest effect.
In contrast, Radicava has a different mechanism of action. It is a free radical scavenger that is believed to relieve the effects of oxidative stress, a likely factor in the progression of ALS. Radicava demonstrated its efficacy in a six-month clinical trial in Japan. At Week 24, patients receiving Radicava declined less on the ALS Functional Rating Scale-Revised (ALSFRS-R), compared to those receiving a placebo. As such, its approval is based on the fact that it makes the patient’s symptoms deteriorate more slowly.
Despite the good news of Radicava’s approval, the drug comes with a heavy price tag. The company stated Radicava would cost $1,087 per infusion, and if taken annually according to the approved dosing and administration, the cost before government discounts will be $145,524.
Ultimately, the holy grail for ALS treatment is still not addressed: disease-modifying drugs that can prevent or halt degeneration of the motor neurons are still needed. Therefore, the pharmaceutical industry should continue to explore the development of drugs with diverse mechanisms of action, as any new addition to the market will be vital for the treatment of ALS patients.