Friday was an incredibly important day. Yes, it was Cinco De Mayo, but it was also the first time the FDA has approved a treatment for ALS (Lou Gehrig’s Disease) in over two decades!
The treatment, edaravone, had already been available in other countries (e.g. Japan, South Korea). The FDA reached out to the Japanese-based company to encourage them to file an application in the US. It will be marketed in the US under the name Radicava, and is being produced by MT Pharma (Mitsubishi Tanabe Pharma America).
The treatment is administered as an infusion. Patients initially would receive the drug daily for two weeks, and then go without treatment for the next two weeks. After the first round of treatment, the infusion days are decreased to 10. In addition, the drug manufacturer has set up a patient support program called Searchlight Support, 1-844-772-4548, that can help determine eligibility, and provide a case manager.
A six-month long clinical trial in Japan consisting of 137 participants showed that patients receiving edaravone had a lesser decline in their daily functioning than those who were receiving a placebo. Edaravone, unfortunately, will not stop the progression of ALS altogether, it will only slow and minimize the effects of the decline.
Severe allergies to the medication are possible, and for those individuals, this medication will not be an option. However, this approval marks growing hope for ALS patients. More research, more data, and more treatments are on the horizon (masitinib is currently under review in Europe).