The FDA was so impressed with Mitsubishi Tanabe's amyotrophic lateral sclerosis drug, it recruited the company to file its data for U.S. approval based solely on data generated in Japan. Now, with a Friday approval for Radicava, ALS patients in the U.S. will have their first new treatment option in 22 years—at the prediscount price of $145,000 per year.
The med doesn't reverse the decline ALS patients suffer, but it has been shown to slow its progression somewhat, according to phase 3 data from a six-month placebo-controlled study in Japan. Patients on the treatment experienced a 33% reduction in the rate of decline in physical function compared with those on placebo.
But ALS patients have few options, and the data were enough to win 2015 approvals in Japan and South Korea. When FDA officials learned about the treatment, they quickly reached out to Mitsubishi Tanabe to request the company file for marketing approval in the U.S., according to an agency release. The Japanese drugmaker submitted its application last June, and has won FDA approval based off its Japanese data. Radicava is expected to be available in the U.S. in August.
Patients with Lou Gehrig’s disease can lose the ability to control their muscles, making it hard to walk, eat, breathe and talk. Most die within two to five years after diagnosis. Radicava is administered intravenously daily for two weeks, followed by 14 drug-free days. For subsequent treatment cycles, treatment entails daily infusion for 10 days within a 14-day period, followed by another two weeks without the medicine.
Speaking with FiercePharma, Joe Palumbo, VP of medical science at MT Pharma America, said the approval is “enormously meaningful” for himself and for the company, as there have been plenty of failed attempts to improve ALS care over the years.
“This is perhaps the biggest day in my career, to know that we have been able to accomplish something this major for this community,” he said.
With each infusion costing $1,086 on a list-price basis, Radicava runs $145,524 per year before any discounts. According to Forbes, the U.S. price is far higher than the company’s $35,000 launch price in Japan, a difference the company attributed to a stroke indication there. That's obviously a much larger market, compared with the rare-disease status of ALS.
Mitsubishi Tanabe says its U.S. price takes into consideration a 13-year development program and other investments, plus the process of building a commercial infrastructure to support its first U.S. launch. The Centers for Disease Control and Prevention estimates there are 12,000 to 15,000 ALS patients in the United States.
Radicava beat AB Science's masitinib and Cytokinetics' tirasemtiv to an FDA approval, both drugs hailed by the ALS Therapy Development Institute as candidates that could work toward an FDA filing soon. AB Science filed for European approval with its med in September, according to ALS Worldwide.
Mitsubishi's drug won approval under the FDA’s orphan drug review program, which provides incentives for drugmakers chasing rare diseases. Patients on the treatment have experienced headaches, problems walking and bruising.
The FDA has been on a roll with drug approvals since the start of the year, now bringing its 2017 approval total to 20. In all of last year, the agency signed off on 22 new meds. Radicava’s approval was the sixth within a 9-day period at the FDA. It's the first ALS drug to notch an approval since Rilutek, brought to market in 1995 by Rhone Poulenc Rorer, now part of Sanofi.