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First new drug to treat ALS in 20 years approved in US

Monday, May 8, 2017,17:07 IST By anju A A A

Washington | The first new drug to treat patients with amyotrophic lateral sclerosis (ALS) in two decades has been approved in the US.

The US Food and Drug Administration (FDA) last week gave approval to Radicava (edaravone) for the treatment of ALS, commonly referred to as Lou Gehrig’s disease.

ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles.

“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 US,” 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,” said Bastings.

In ALS, voluntary muscles produce movements such as chewing, walking, breathing and talking.

The nerves lose the ability to activate specific muscles, which causes the muscles to become weak and leads to paralysis. ALS is progressive, meaning it gets worse over time.

The US Centers for Disease Control and Prevention estimates that about 12,000-15,000 Americans have ALS. Most people with ALS die from respiratory failure, usually within three to five years from when the symptoms first appear.

Radicava is an intravenous infusion given by a health care professional, FDA said.

It is administered with an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period. Subsequent treatment cycles consist of dosing on 10 of 14 days, followed by 14 days drug-free.

The efficacy of edaravone for the treatment of ALS was demonstrated in a six-month clinical trial conducted in Japan.

In the trial, 137 participants were randomised to receive edaravone or placebo.

At week 24, individuals receiving edaravone declined less on a clinical assessment of daily functioning compared to those receiving a placebo, according to FDA.

The most common adverse reactions reported by clinical trial participants receiving edaravone were bruising (contusion) and gait disturbance.

Radicava is also associated with serious risks that require immediate medical care, such as hives, swelling, or shortness of breath, and allergic reactions to sodium bisulfite, an ingredient in the drug, FDA said.