FDA recently approved the first new treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, in more than 20 years.
However, edaravone (Radicava, Mitsubishi Tanabe Pharma America), will cost more than $145,000 annually, according to the manufacturer.
“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, MD, deputy director of the Division of Neurology Products in FDA’s Center for Drug Evaluation and Research, in a statement from 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.”
Related: Novel drug approved for Huntington's symptom
Radicava’s price tag “represents the investments MT Pharma America is making to deliver it to U.S. patients” as a mid-stage orphan drug, the company said in a statement. However, MT Pharma America says it will offer co-pay assistance for commercially insured patients to help reduce their out-of-pocket costs.
The Centers for Disease Control and Prevention estimates that approximately 12,000 to 15,000 Americans have ALS. Most people with ALS die from respiratory failure, usually within 3 to 5 years from when the symptoms first appear.
Radicava, an intravenous infusion given by a healthcare professional, has 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.
Related: New Parkinson's drug, 2 others approved
The efficacy of the drug, which was granted orphan designation, was demonstrated in a 6-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.
Related: First drug for rare skin cancer wins approval
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, according to FDA. Sodium bisulfite may cause anaphylactic symptoms that can be life-threatening in people with sulfite sensitivity.