FDA approves first new treatment for ALS in two decades
pharmafile | May 8, 2017 | News story | Sales and Marketing | MLS, Mitsubishi Tanabe Pharma America, Radicava
The FDA has approved only the second form of treatment for amyotrophic lateral sclerosis (ALS), with the previous drug, riluzole, having been approved two decades ago. The new treatment is called Radicava (edaravone) and is produced by Japanese company, Mitsubishi Tanabe Pharma America; it has already been approved for use in Japan for the past two years.
The treatment was found, in a six month trial conducted in Japan, to slow progress of the disease. The study revealed that, compared with patients receiving placebo, those individuals who received treatment with Radicava declined less on a clinical assessment of daily functioning.
ALS is a progressive disease that sees the loss of control of muscles that control activities such as walking, talking, breathing and eating. Once those suffering with the condition are diagnosed, most do not live beyond five years. Stephen Hawking is the one of the most well-known sufferers, who continues to defy the prognosis that he would not live very long after being diagnosed.
ALS awareness was increased dramatically by the ‘ALS Ice Bucket Challenge’ that swept social media in 2014, and intermittently ever since. The campaign went on to raise approximately $115 million to research into the disease.
Radicava was originally approved by Japanese regulators in 2015. It is administered through intravenous injection in cycles; with 14 injections in 14 days, followed by two weeks without treatment before recommencing doses.
“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 treatment will not come cheap, with the list price of the treatment over 12 months coming to $145,523. This is far higher than the price paid in Japan for the treatment, where it costs $35,000 per year. It is estimated that there are 15,000 patients with ALS currently in the US who can now be offered a new form of treatment to slow the disease’s progression.
Ben Hargreaves
