FDA on Friday approved Mitsubishi Tanabe Pharma's Radicava to treat the neurological disorder amyotrophic lateral sclerosis (ALS).
Radicava is only the second ALS treatment FDA has approved for sale in the United States. According to the New York Times, FDA approved the first treatment, called Riluzole, for ALS more than 20 years ago.
ALS is a rare progressive condition that affects an estimated 12,000 to 15,000 U.S. residents, according to CDC. ALS attacks and kills the nerve cells that control voluntary muscles used to breathe, chew, talk, and walk.
The affected nerves eventually lose the ability to activate certain muscles, which leads to muscle weakness and paralysis, according to FDA. In most cases, people with ALS die from respiratory failure within three to five years of when they first experience symptoms of the disease.
FDA approved Radicava after taking the unusual step of asking Mitsubishi Tanabe Pharma to seek regulatory approval for the drug in the United States. Radicava has been sold in Japan and South Korea since 2015, Reuters reports.
According to FDA, the agency approved Radicava based on the results of a six-month clinical trial, which was conducted in Japan and involved 137 participants. Participants with ALS who received Radicava for six months experienced a 33 percent reduction in their rate of decline when compared with those who received a placebo. FDA did not require clinical trials to be conducted in the United States in an apparent move to speed Radicava's approval, MedCity News reports.
MT Pharma America (MTPA), a subsidiary of Mitsubishi Tanabe Pharma Corporation, on Friday said Radicava will cost $145,524 for a year's worth of treatment, about or $1,086 per infusion. MTPA Chief Commercial Officer Tom Larson said the drug should be available in the United States by August.
Patient advocacy groups applauded Radicava's approval.
Barbara Newhouse, president and CEO of The ALS Association, in a statement thanked "FDA and MT Pharma for working together to expedite the approval of the first new ALS-specific treatment in decades."
Neil Shneider, director of the Eleanor and Lou Gehrig ALS Center at Columbia University Medical Center, said he is "very happy, frankly, that there is a second drug approved for ALS," adding that the drug's "effect is modest but significant."
However, Shneider said he might hesitate to prescribe the treatment because its method of administration, which occurs via intravenous infusion over periods of 10 to 14 days, can be difficult. "If it were a pill and easily administered, I'd be less reluctant to use it," he said (Grover, Reuters, 5/5; Fiore, MedPage Today, 5/5; Burton, Wall Street Journal, 5/5; Preston, MedCity News, 5/5; Grady, New York Times, 5/5; ALS Association statement, 5/5; FDA release, 5/5).
Pharmacy leadership roles: Results and analysis from the 2017 Pharmacy Leaders Survey
Regina Lohr, Senior Consultant
As the pace of change in health care has accelerated, pharmacy services have had to evolve at an increasingly rapid pace. Yet pharmacy leadership roles and organizational structures are in many cases stuck in the past.
To better understand the pharmacy leadership landscape, the Pharmacy Executive Forum conducted a national survey on early 2017. Join us as we provide an analysis of the results.