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New Drug Edaravone Approved for Treatment of ALS may have Additional Health Benefits in Stroke and Nephropathy

Susanna Sisson's picture
IV treatment

The drug Radicava (edaravone), manufactured by the Japanese company Mitsubishi Tanabe Pharma Corporation (MT Pharma), a subsidiary of Mitsubishi Chemical Holdings Corporation has been approved by the Food and Drug Administration (FDA) for treatment of Amyotrophic Lateral Sclerosis (ALS) in the United States. Edaravone is the first drug for treatment of ALS to be developed and approved since riluzole (Rilutek) was approved in December 1995.


The approval of edaravone follows the release of data presented at the 27th International Symposium on ALS/MND in Dublin, Ireland in December, 2016. The Phase III study of the drug lasted 48-weeks and included concomitant treatment with standard of care drug riluzole (Rilutek). Participants were patients with ALS or probable ALS with a diagnosis of less than two years. A 24-week double blind placebo controlled phase was followed by a 24-week open label treatment phase. [1]

The criteria for entering the study not only included early diagnosis but minimal loss of function, the ability to perform activities of daily living, and normal respiratory function.

Patients were assessed using the ALS Functional Rating Scale-Revised (ALSFRS-R). The patients who received 48 weeks of intravenous edaravone treatment in 10-14 day cycles exhibited significantly less functional loss especially with regard to motor and respiratory function decline. There was virtually no difference in serious side effects between the group receiving edaravone and the placebo group even in those patients who received edaravone for the full 48 weeks and there were no deaths associated with the study. [1][2]

Joseph M. Palumbo, MD, Vice President, Medical Sciences and Translational Research, Mitsubishi Tanabe Pharma Development America, Inc. explains "These findings suggest that intervention with edaravone may provide a treatment option to people living with ALS when therapy is promptly initiated."

About ALS

ALS is a rare degenerative neurological disease that destroys motor neurons in the brain and spinal cord resulting in loss of motor function, resulting in eventual paralysis and death. The incidence of ALS is slightly higher in men than women and usually diagnosed between the ages of 55-75. ALS does not generally affect bladder function or sensory function of taste, smell, touch, hearing or sight. The disease is painless yet debilitating. Initial symptoms are insidious and may be overlooked as normal aging or mistaken for other diseases such as thyroid disease, multiple sclerosis (MS), stroke, or transient ischemic attack (TIA). Symptoms [3] may include loss of hand strength and ability to grip objects or weakness in lower limbs, muscle cramps or twitching, tripping, difficulty swallowing, a change in voice pitch, slurred speech, or periods of uncontrollable laughing or crying. As the disease progresses muscles of the respiratory system are affected leading to paralysis and eventual need for artificial ventilation and complete care. The average life expectancy is three to five years although some patients do live five years or longer and some have lived up to ten years. Those with familial ALS, which accounts for about 5-10% of cases, have a lower survival rate of 1-2 years. One of the most famous people with ALS is Stephen Hawking who has a slow progressing form of the disease and is now 75 years old.

Differential diagnosis of ALS includes:

• Physical exam and assessment including full neurological workup
• Blood and urine tests including biomarkers for oxidative stress, comprehensive thyroid and parathyroid levels, serum protein electrophoresis and 24-hour urine for heavy metals.
• Spinal tap
• X-rays and magnetic resonance imaging (MRI)
• Electromyography (EMG) and nerve conduction velocity (NCV)
• Myelogram of cervical spine
• Muscle and or nerve biopsy

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Edaravone mechanism of action and applications

Oxidative stress is a known factor in ALS. Edaravone is a potent antioxidant which is thought to have neuroprotective effect on the neurons of the brain and spinal cord by scavenging free radicals and suppressing nitration of tyrosine residues in the cerebrospinal fluid. [1][2][4]

Edaravone has successfully been used in stroke patients to decrease damage and improve patient outcomes. Results of another study in China published in Neural Degenerative Research hypothesize that edaravone reduces oxygen-glucose-serum deprivation/restoration (OGSD/R) induction of apoptosis of spinal cord astrocytes. [5] In other words, edaravone reduces cell death of neurons.

There are a myriad of studies with regard to edaravone’s effect on prevention of cellular damage due to oxidative stress, including studies involving Parkinson’s and Alzheimer’s disease, drug-induced nephropathy and cerebral toxicity, ischemic stroke and post-operative ischemia, pneumococcal meningitis, diseases such as macular degeneration, and even peritoneal sepsis. Truly the list of potential applications with regard to combating oxidative stress seems limitless.

Cost of Edaravone

While edaravone may limit cellular damage, the cost of the drug is prohibitive in many potential applications. In the treatment of ALS, the cost is $1000 per infusion which adds up to a yearly cost of nearly $150,000. However, MT Pharma says it will offer co-pay assistance for insured patients and that it has developed a program to help uninsured patients who meet certain requirements.

The recommendation for patients on edaravone is simultaneous treatment with riluzole. Out of pocket cost for those without insurance for riluzole is about $400 for thirty 50mg tablets. [6]

The approval of edaravone gives new hope to patients with ALS. The drug is expected to be available in the U.S. in August 2017.