New Products

Mitsubishi Tanabe wins the race to snag the first new ALS drug OK in more than 20 years

After being recruited by the FDA, Mitsubishi Tanabe has won the agency’s approval to start selling edaravone as a new treatment for ALS — the first new drug for this lethal disease in more than two decades and only the second now available to patients.

Regulators asked the Japanese pharma company to file the drug for ALS after its approval in Korea and Japan. The drug — which has also been sold for acute ischemic stroke for years in Japan — will now hit the market as Radicava.

For a short time at least, a new subsidiary group set up to sell the drug will have a clear crack at a market that was slated to shrink as the sole branded ALS drug lost revenue to generics. The last new therapy, Rilutek (riluzole) from Sanofi, arrived in the US in the mid-90s, underscoring just how difficult this disease has been to tackle in the clinic.

Radicava is no cure. Patients continued to decline in the 167-patient study reviewed by the FDA, but they weren’t so quick to lose their ability to function. And in a twist, it arrives after regulators asked the pharma company to file for an OK.

Eric Bastings, FDA CDER

“After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States,” said Eric Bastings, deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option.”

The company hasn’t formally distributed data on the drug, that’s coming up at a scientific conference later in the year, but researchers have noted that patients taking a 60 mg dose of the drug declined at a rate roughly a third slower than the placebo arm, comparing them to baseline on the revised ALS Functional Rating Scale.

Neil A. Shneider, director of the Eleanor and Lou Gehrig ALS Center at Columbia University Medical Center, told The New York Times that the “effect is modest but significant.”

Mitsubishi Tanabe is planning to go after the market with a price of $1,086 per infusion and an annual list price of $145,524, which could pose some trouble for the pharma company. In addition to the high cost and limited benefits, Shneider and others in the field are none too happy about the risks posed to patients who will need to keep an IV line left in place for their infusions, raising the risk of infection.

In the meantime, there are at least two other biotechs looking to make their mark in the field. Cytokinetics is one, with a late-stage drug called tirasemtiv. AB Science’s AB-1010, an oral tyrosine kinase inhibitor, was filed for an approval in Europe last fall.


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RAPS Regulatory Convergence 2017