- The Food and Drug Administration on Thursday approved a new version of Mitsubishi Tanabe Pharma's Radicava, clearing the way for a likely more convenient treatment option for patients with amyotrophic lateral sclerosis.
- This version, called Radicava ORS, is taken orally as opposed to the original, which is administered via an hourlong infusion. With both forms, patients are supposed to receive daily doses for about a third of each month, followed by two weeks without the drug. Mitsubishi noted in a statement how Radicava ORS offers a "flexible" option that "may take only a few minutes to administer."
- Radicava was first approved as a treatment for ALS — better known to some as Lou Gehrig's disease — in 2017. In its last fiscal year, which ended March 31, Mitsubishi said sales from the drug totaled 24.6 billion yen, or roughly $190 million at current exchange rates.
ALS is a progressive illness that erodes nerve cells, leading to the loss of essential functions like walking, eating and breathing. It's also fatal. After diagnosis, patients live on average just two to five years.
Like many diseases of the brain and nervous system, the complex biology of ALS has time and again thwarted efforts to develop effective medicines against it. Nearly a century after Lou Gehrig was diagnosed, there are only two main drugs approved to treat ALS, and both have limitations.
One of those drugs, an oral molecule named riluzole, was first cleared by the FDA in 1995 and has been shown to prolong survival by a few months. Merit Cudkowicz, director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, told BioPharma Dive last year that about four in five U.S. patients take riluzole because of this benefit and the drug's lack of harsh side effects.
Radicava, meanwhile, was approved based on results from a study of about 140 participants, in which the drug appeared to have a modest effect on the rate at which patients' functions declined.
But that limited effect, coupled with the many hourlong infusions the drug requires, has hindered its use. Cudkowicz said that among the U.S. ALS population, which is estimated to total 15,000 to 30,000 patients, far fewer receive Radicava compared to riluzole.
Mitsubishi is now looking to change that with the introduction of Radicava ORS.
Patients often prefer oral drugs to those that are injected or infused. They're usually quicker to take, and can be administered at home rather than by a healthcare professional. In the case of Radicava ORS, Mitsubishi also notes the new formulation can be given through a feeding tube and doesn't require refrigeration and reconstitution before taking.
Another oral ALS drug may soon come to market, with the FDA expected to make an approval decision by June 29. That drug, developed by Amylyx Pharmaceuticals, showed modest signs of both slowing functional decline and extending survival in a clinical trial similar in size to the one that led to Radicava's approval.
Despite those results, it's unclear whether Amylyx's drug will get the go ahead. FDA staff have taken issue with the way the company designed its study and analyzed its results. And in March, a group of outside experts who advise the agency on brain drugs ultimately voted, by a slim margin, that there wasn't enough evidence to support the drug's approval.