The FDA has approved edaravone (Radicava, Mitsubishi Tanabe Pharma America, Inc.) for the treatment of patients with amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease. In the United States, the only other approved ALS medication, generic riluzole, modestly slows disease progression in some patients.
Edaravone is expected to be available in the U.S. by August. Mitsubishi Tanabe has priced the treatment at $1,086 per infusion. If taken annually for 12 months or 13 cycles, according to the dosing and administration included in the label, the total cost before government discount would be $145,524.
Edaravone is an intravenous infusion administered by a health care professional. 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 study, 137 participants were randomly assigned to receive edaravone or placebo. At week 24, individuals treated with edaravone declined less on a clinical assessment of daily functioning compared with those receiving placebo.
The most common adverse events reported by clinical trial participants receiving edaravone were bruising and gait disturbance.
Edaravone is also associated with serious risks that require immediate medical care, such as hives, swelling, and 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 patients with sulfite sensitivity.
ALS 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. The Centers for Disease Control and Prevention estimates that approximately 12,000 to 15,000 people have ALS in the U.S. Most people with ALS die from respiratory failure, usually within three to five years after the symptoms first appear.
Sources: FDA(link is external); May 5, 2017; and Reuters(link is external); May 5, 2017.
RWE is a relatively new kid on the block. How exactly it will fit into the complicated world of cancer drug testing, approval, regulation, and marketing is uncertain. The randomized clinical trial has been the gold standard in oncology research for decades and will remain so for the foreseeable future.
American Telemedicine Association
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American Cancer Society
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Neil Minkoff, MD
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