FDA clears new ALS drug for US market
- The Food and Drug Administration has approved Radicava (edaravone) in the U.S. as the first new drug in two decades to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. This will be marketed by MT Pharma America, a division of Japanese company Mitsubishi Tanabe Pharma.
- Radicava’s price tag is expected to be $145,000 in the U.S., significantly higher that its original price in Japan, which is priced there at about $35,000 per year.
- Radicava is given as an intravenous infusion daily for 14 days, followed by a 14-day gap; subsequent treatment is dosing on 10 of 14 days, followed by 14-day gaps.
The arrival of Radicava for ALS marks the first new drug in the market for this rare disease since 1995, when the Food and Drug administration approved the disease-modifying agent Rilutek (riluzole). While Radicava doesn’t affect the course of the disease, it does slow symptoms and offer additional choices.
“For people with ALS and their families, having a new therapy which slows the decline of physical ability is incredibly significant,” said Jonathan S. Katz, ALS Clinic Director, Forbes Norris MDA/ALS Research and Treatment Center at California Pacific Medical Center.
However, the new treatment choice does come with a hefty price tag, and provides only a 33% buffer from decline of physical function. Following approval in 2015, Radicava retails for $35,000 in Japan, where all prescription drug prices are reviewed and potentially cut each year. The U.S. price is expected to be $145,000 a year, more than four-fold higher.
Atsushi Fujimoto, president of MT Pharma America, acknowledges the high cost. "We recognize how important this therapy may be to people with ALS and are committed to helping provide access to this important treatment option, with the goal of keeping out-of-pocket costs at a minimum for eligible patients," he said.
The U.S., unlike Japan, does not have regulated drug costs and the high price tag will allow the company to recoup some of the costs it had to shell out to develop the drug. The argument can also be made that the drug is serving two different markets —Radicava is approved for treatment of stroke in Japan, a large patient population, while ALS is a rare disease.
Radicava has orphan drug status in both the U.S. and Europe. ALS is a rare but rapidly progressing neurodegenerative disease that causes death within two to five years. There are around 5,000 to 6,000 new cases each year in the U.S.
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