Drugmaker Wins FDA Approval for First New ALS Drug in Decades

  • Radicava slowed decline in six-month study of 137 patients
  • Intravenous medicine will cost about $145,000 a year in U.S.

Mitsubishi Tanabe Pharma Corp. won U.S. approval for its medication to treat Lou Gehrig’s disease, the first new drug for the condition in more than two decades and just the second approved option for patients with the rare and deadly muscle-weakening ailment.

Radicava will cost about $145,000 a year before any discounts and rebates, with assistance available to help insured patients cover co-payments, the company said in a statement. The intravenous medication was approved based on a six-month study of 137 patients in Japan with amyotrophic lateral sclerosis, or ALS, after it slowed their physical decline.

The disease is marked by the inexorable loss of control over the voluntary muscles that control functions like breathing, walking, talking and eating. It’s diagnosed in about 5,000 Americans each year, according to U.S. government data, and the Centers for Disease Control and Prevention estimates that there are 12,000 to 15,000 Americans living with the condition. Most people die within three to five years of the appearance of symptoms, according to the U.S. Food and Drug Administration.

In an unusual twist, the FDA reached out to the Japanese company after hearing about its work on the therapy. The agency “rapidly engaged” with Mitsubishi Tanabe about filing a marketing application in the U.S., Eric Bastings, deputy director of the FDA’s division of neurology products, said in a statement.

“This is the first new drug approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option,” he said. The drug joins Rilutek, originally developed by Sanofi, for treating the disease.

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