The new FDA-approved oral formulation of Radicava ORS (edaravone) is more convenient than the intravenous formulation, yet the efficacy and composition remain the same. The oral formulation may also be given through a feeding tube without the need for dose adjustments.
Radicava ORS, the original intravenous formulation, was approved by the FDA based on its ability to slow the loss of physical function caused by amyotrophic lateral sclerosis (ALS). According to a recent article in Biospace, the disease became known in the 1940s as Lou Gehrig’s disease named after the famous ballplayer who retired at the height of his career due to ALS.
Another famous ALS patient was British theoretical physicist, cosmologist, and author Stephen Hawking. Dr. Hawking, born in 1942, was one of only ten percent of ALS patients who lived well past the five-year survival period. He died in 2018.
Treatment Cycles and Intervals
At the start of the treatment, the patient is required to fast overnight. The patient takes the drug the following morning either in oral form or through a feeding tube. Radicava ORS is administered daily for fourteen days. Then the patient has a fourteen-day drug-free interval at which time the cycle is repeated. However, in this case, the drug is given only ten out of the fourteen days. The next drug-free interval remains the same as the last.
Worldwide Approval
Mitsubishi Tanabe Pharma and MTPC developed Edaravone to slow the progression of ALS. As of 2021, six thousand five hundred patients in the United States have been treated with the drug. Japan and South Korea approved Radicava for ALS in 2015. Between 2018 and 2021 six countries, including Canada, approved marketing authorizations for Radicava.
The FDA based its approval on favorable results from a randomized clinical trial involving 137 patients. The participants in the trial received either a placebo or Radicava ORS.
Using the ALS Rating Scale, measurements at week twenty-four of the trial showed the participants had thirty-three percent less loss of function. This measurement was compared against the placebo group.
About Amyotrophic Lateral Sclerosis (ALS)
ALS, a rare and progressive neurological disease, primarily affects nerve cells that control voluntary muscle movement.
ALS is part of a large group of disorders called motor neuron diseases. Motor neurons (nerve cells) start in the brain and extend to the spinal cord and muscles in all areas of the body. As motor neurons malfunction, the brain does not receive signals and can no longer control voluntary movements.
The majority of patients do not survive more than five years after diagnosis. To date there has been no cure for ALS nor has there been any effective treatment to slow or halt the disease.
The NIH is investing $25 million over a five-year period in a program for innovative ALS research. Approximately 16,000 people in the U.S. have ALS. JourneyMate Support has been launched by MTPA to provide resources to assist ALS patients and caregivers.
