The U.S. Food and Drug Administration (FDA) announced on May 5 that the intravenous drug Radicava, developed by Japan-based Mitsubishi Tanabe Pharmaceuticals, has been approved for the treatment of amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease.
Radicava, with the generic name edaravone, is the first and only ALS treatment drug approved by FDA since Rilutek, which was approved in 1995.
What Is Radicava?
Radicava is an intravenous drug that is clinically tested and manufactured in Japan for the treatment of stroke. However, medical practitioners have seen the drug's effectiveness in slowing down the symptoms of ALS and have been prescribing it to ALS patients, as well.
The drug went through multiple Phase 3 trials, including a six-month clinical trial wherein 137 ALS patients participated. According to the results, the 69 ALS patients who received Radicava experienced 33 percent less decline in physical functions. This translates to 2.49 points in the ALS Functional Rating Scale-Revised (ALSFRS-R).
The results of the clinical trials in Japan led the FDA to approve Radicava without requiring the pharmaceutical company to launch clinical trials in the United States.
"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," Dr. Eric Bastings said. Dr. Bastings is the deputy director of the Division of Neurology Products in FDA's Center for Drug Evaluation and Research.
Radicava Treatment And Price
Treatment is given in 28-day cycles wherein a patient would receive Radicava daily or near daily for 14 days, followed by a 14 day break from the drug. Of course, Radicava works best on patients who are still in the early stages of ALS.
While this is good news for families of ALS patients, it comes at a steep price because the MT Pharma America, the company's arm in the United States, projects the annual cost for patients at $145,524 before government discounts. This is a pretty steep price considering Radicava's launch price in Japan was at $35,000.
The good news is that the company is offering to help ALS patients with the financial obligation.
"We will offer co-pay assistance for commercially insured patients to help reduce their out-of-pocket costs," the company expressed.
Possible Side Effects
The more serious side effects of Radicava include hypersensitivity reactions, especially with allergies, and sulfite allergic reactions, shortness of breath, swelling, and hives. More common side effects include headaches, bruising, and gait disturbance.
Radicava will be manufactured in Japan so MT Pharma America estimates that the first batch of treatments will only arrive in the United States by August 2017, but patients and healthcare providers can stay updated on its status by signing up for updates in the Radicava website.
Just to emphasize, Radicava is not a cure. It will not extend the life of the patients the drug is administered to but it will slow down the progression of the disease. ALS remains incurable and is always fatal — at least until science finds a way.