A new treatment option for patients with amyotrophic lateral sclerosis (ALS) is now available in US pharmacies for the first time in 22 years. Radicava (edavarone), developed and manufactured by Mitsubishi Tanabe Pharma America, was approved by the US Food and Drug Administration (FDA) in May 2017. It was granted orphan drug designation, which provides incentives to promote, assist and encourage the development of drugs for rare diseases and medical conditions.
ALS is an incurable neurodegenerative disease that affects the nerve cells in the spinal cord and brain and eventually results in a loss of ability to initiate and control muscle movement. It can lead to paralysis and death within two to five years of diagnosis. The Centers for Disease Control and Prevention (CDC) estimates up to 15,000 people in the US have ALS, and that up to 5,000 people will be diagnosed with the condition each year. The causes of the condition are unknown, but between five and ten percent of cases occur within families.
Radicava is an intravenous infusion administered by a healthcare professional at an ALS center, free-standing infusion center, hospital outpatient department, physicians office or through a home infusion provider. The efficacy of the drug was shown in a clinical trial, which demonstrated Radicava slowed the rate of loss of physical function in ALS patients by 33 percent.
The quick approval of Radicava and the speed with which it has been made available in US pharmacies was welcomed by The ALS Association, which commended the FDA and Mitsubishi Tanabe Pharma America for cooperating to expedite approval of the drug. The FDA approved Radicava less than one year after a New Drug Application was submitted by Mitsubishi Tanabe Pharma America.
"We hope [the] announcement signals the beginning of a new chapter in the fight against this terrible disease. There are several drugs to treat ALS currently in clinical trials and we are hopeful that people living with ALS have even more therapies available to them sooner rather than later," said Barbara Newhouse, president, and CEO of The ALS Association.
The last treatment approved specifically for ALS was Rilutek (riluzole), developed by Sanofi, which was approved in 1995. It is available in tablet and liquid form and works to delay the onset of ventilator-dependence in selected patients.
For more information on Radicava, follow the link below:
https://www.radicava.com/patient/
