The U.S. Food and Drug Administration has approved use of a medicine proved to slow the degenerating effects of the rare disease ALS.
It’s the first time in more than two decades the agency approved a treatment for the progressive disease amyotrophic laterals sclerosis – also known as Lou Gehrig's disease -- which affects 12,000 to 15,000 people in the United States.
On Friday, the FDA gave its OK to Mitsubishi Tanabe Pharma America for the drug Radicava, or edaravone. The drug is expected to be available by August.
The FDA said the approval came after a Japanese study displayed the drug’s benefits.
The six-month trial tested more than 100 people and found those who used edaravone “declined less” in terms of daily functioning opposed to those who did not use the drug.
Dr. Eric Bastings, the deputy director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research, said the agency quickly encouraged the drugmaker to file to make the drug available in the U.S.
“This is the first new treatment approved by the FDA for ALS in many years,” Bastings said, “and we are pleased that people with ALS will now have an additional option.”
Previously, the only drug approved by the FDA to treat ALS was Rilutek, according to the Mayo Clinic. Rilutek, or riluzole, was granted FDA approval in 1995.
ALS kills nerve cells connected to voluntary muscles. The disease, which gets worse over time, could inhibit vital movements such as chewing, breathing, talking and walking. The FDA said muscles eventually become weak and a person usually dies from respiratory failure with 3-5 years.
Radicava would be given by infusion for a period of two weeks to start, followed by a 14-day period without drugs. The cycles then continue in two-week intervals, followed by periods without the drug.
Radicava does have side effects. The FDA said trial participants experienced bruising and problems walking. The drug also has caused hives, swelling, shortness of breath and allergic reactions.
The ALS Association states Radicava “is not a cure” but may help people live with the disease. It said the going price for Radicava is $11,000 per dosage cycle, or about $146,000 a year.
However, MT Pharma America said it will provide the drug at no cost for those without insurance and who meet certain requirements.
Follow Sean Rossman on Twitter: @SeanRossman