Edaravone at ALS

On May 5th, 2017, the Food & Drug Association (FDA) approved the drug Edaravone for the treatment of amyotrophic lateral sclerosis (ALS) in the USA; it had previously been approved in Japan. Edaravone is also approved in Switzerland. A single import is possible; You can apply for reimbursement from the health insurance company.

Treatment with Edaravone is via intravenous infusions. This involves a cycle of therapy in which one infusion is given daily for 10 to 14 days, followed by a 14-day break before the next infusion phase begins. The drug is essentially well tolerated; a survey conducted in 2017/2018 on the satisfaction of ALS patients with the preparation was positive. The mechanism of action of edaravone is not well understood - it is currently assumed that it has a so-called antioxidant and thus neuroprotective effect.

Since previous studies on patients have only been carried out in Japan, there is the possibility of a changed effectiveness of the drug in a central European patient cohort. The first placebo-controlled randomized study published in 2015 (classic approval study) could not demonstrate any positive effect of edaravone in ALS. The authors concluded at the time that this does not justify an indication for use in ALS. In the meantime, two follow-up studies have been carried out that included specific subpopulations of ALS sufferers: although no superiority of the drug compared to placebo could be demonstrated in advanced ALS patients, patients in a defined group (overall not yet severely affected, breathing hardly affected , disease duration no longer than 2 years) showed a slowing of progression compared to the placebo controls. It has not yet been clarified to what extent the simultaneous administration of riluzole, which is currently approved in Germany, influences the effectiveness of edaravone.

On May 29, 2019, the European Medicines Agency (EMA) published that the application for approval for Radicava had been withdrawn by Mitsubishi Tanabe Pharma GmbH. This was based on the preliminary assessment of the EMA, according to which the current study situation does not allow approval and further data are necessary. It remains to be seen what this means for everyday use. The procedure with individual applications at the health insurance fund remains unchanged, but increasing rejection must be expected. We advise every insured person and practitioner to have the assumption of costs checked now in order to avoid possible recourse claims.