ALS Treatment Drug Approved by FDA
Radicava has been approved to treat patients with ALS, commonly referred to as Lou Gehrig's disease.
The FDA announced it has approved Radicava to treat patients with amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease.
Radicava is an intravenous infusion administered with an initial treatment cycle of daily dosing for 14 days.
"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, M.D., deputy director of the Division of Neurology Products in 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 drug's efficacy was demonstrated during a six-month trial in Japan where 137 participants were randomized to receive edaravone or placebo. At Week 24, individuals who given Radicava declined less on a clinical assessment of daily functioning than those receiving a placebo, according to FDA's announcement.
FDA granted the drug orphan drug designation, which provides incentives to assist and encourage development of drugs for rare diseases. The agency granted approval of Radicava to Mitsubishi Tanabe Pharma America, Inc.
The most common adverse reactions reported by clinical trial participants receiving the drug were bruising and gait disturbance. The announcement says it is 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. Sodium bisulfite may cause anaphylactic symptoms that can be life threatening in people with sulfite sensitivity.