Mitsubishi Tanabe scores FDA nod for oral version of its ALS drug Radicava

Hour-long infusions at a clinic, 10 days a month: Patients with amyotrophic lateral sclerosis (ALS) have had to endure an onerous treatment regimen.

But on Thursday, the FDA signed off on an oral version of Mitsubishi Tanabe’s Radicava, which can be taken at home and, if necessary, through a feeding tube.

When Radicava was initially approved in 2017, it was the first new treatment in 22 years for ALS—also known as Lou Gehrig’s disease. While the condition is fatal, usually two to five years from diagnosis, Radicava has been shown to slow its progression by roughly a third. Patients with ALS lose the ability to control their muscles, making it difficult to walk, eat, breathe and talk.

The new FDA endorsement is based on evidence showing that the oral version can deliver the same concentration of medicine to the bloodstream.  

The oral Radicava is taken in the morning on an empty stomach and is administered with the same frequency as the infused version. The drug is introduced into the body with doses for 14 straight days, followed by 14 drug-free days. For maintenance, patients take it 10 out of 14 days, followed by 14 days with no medicine.

After Radicava won approval in Japan and South Korea in 2015, the FDA reached out to Mitsubishi Tanabe to apply for an orphan drug nod in the United States. The blessing came less than a year after submission and was based solely on a successful trial of the drug in Japan.

Radicava also is used in Japan to reduce the risk of stroke.

There are 12,000 to 15,000 people in the U.S. with ALS. Those who take Radicava can have side effects such as bruising, headache and gait disturbance.

Another promising ALS candidate, from Amylyx, could be approved in the U.S. later this year. The drug has gained support from advocacy groups and drawn attention from industry watchers who have compared its hasty run through the regulatory process to that of Biogen’s troubled Alzheimer’s treatment Aduhelm.