May 8, 2017Regulatory
Amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig's disease, is a rare disease that attacks and kills the nerve cells that control voluntary muscles. 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 Centers for Disease Control and Prevention estimates that approximately 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.
The FDA has approved granted approval of Radicava (edaravone) to Mitsubishi Tanabe Pharma America, Inc. to treat patients with ALS. Radicava is administered with an initial treatment cycle of daily intravenous infusions 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 was demonstrated in a six-month clinical trial conducted in Japan. In the trial, 137 participants were randomized 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. 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. Sodium bisulfite may cause anaphylactic symptoms that can be life-threatening in people with sulfite sensitivity.
The FDA granted this drug orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.