US FDA approves first ALS drug treatment in over 20 years

May 8, 2017

The US Food and Drug Administration (FDA) has approved a new drug to treat the fatal neurological disorder ALS, becoming the first such regulatory approval in the US in more than 20 years.

Amyotrophic lateral sclerosis (ALS), sometimes known as Lou Gehrig’s disease, is a rare condition that affects between 12,000 and 15,000 Americans, according to the US Centers for Disease Control and Prevention (CDC).

The new drug – which is chemically known as edaravone and will be sold under the brand nameRadicava – was developed by Japanese pharmaceuticals company Mitsubishi Tanabe Pharma Corporation. Although the drug is not a cure, it can slow down the inevitable worsening of the disease, which gradually paralyzes patients completely. There is still no cure for the disease.

“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 Dr. Eric Bastings, deputy director of the Division of Neurology Products at FDA.

“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,” he said. It is the first new drug approved for ALS since 1995, when riluzole, sold under the brand name Rilutek, was approved.

Radicava is given in the form of an intravenous infusion, with two weeks of daily treatments followed by a two week break.

Tests on a very small group of 137 patients showed those who got the drug had slower declines compared to those who did not.

Mitsubishi Tanabe Pharma America says the drug will cost US $1,086 per infusion.”If taken annually for 12 months or 13 cycles, according to the dosing and administration in the label, the cost before government discounts, will be US$145,524,” the company said.

“We will offer co-pay assistance for commercially insured patients to help reduce their out-of-pocket costs,” the company added. “The price of Radicava is at the mid-point for an orphan drug in the US and represents the investments MT Pharma America is making to deliver it to US patients.”

An orphan drug is one that is unlikely to have many buyers, so the FDA speeds approval.

Another promising drug for ALS is being developed by French drugmaker AB Science SA, which in March reported positive late-stage data on its drug, masitinib. The drug is now under European review.


Category: Features, Pharmaceuticals

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