Mitsubishi Tanabe Pharma Corporation (MTPC), which is locally based in Jersey City, N.J. with a head office in Osaka, Japan, announced yesterday that a New Drug Application has been submitted to the U.S. Food and Drug Administration (FDA) for edaravone (MCI-186) for the treatment of amyotrophic lateral sclerosis (ALS).

Those in the ALS community may have questions about what this means for them. Below we provide some background information on edaravone.

What is edaravone?

Edaravone is believed to act as a free radical scavenger, a compound that works by getting rid of toxic waste generated as a normal by-product of cell function. In ALS it is thought that these by-products are not as effectively removed and the compound may be neuroprotective by relieving the effects of this oxidative stress. The increase in oxidative stress is thought to damage motor neurons (cells that die in ALS).

History of edaravone

Originally, edaravone was approved in Japan to treat stroke. A phase III double-blind placebo-controlled study (MCI186-J16) was conducted in people living with ALS to confirm safety and efficacy of edaravone. This study showed that edaravone did not significantly differ from placebo using the ALS Functional Rating Scale Revised (ALS-FRS-R). Investigators next conducted another phase III trial with a sub-group of people living with ALS that had greater baseline functionality. Results demonstrated that people living with ALS that had a forced vital capacity of >80% and had disease less than 2 years showed the greatest benefit with a statistically significant improvement in the (ALS-FRS-R). It was also established in an exploratory study that edaravone has no benefit from treatment in more advanced ALS cases.

In 2015, edaravone was approved for use as a treatment for ALS in Japan and South Korea using intravenous injections of the drug. In the same year, the FDA and European Commission granted Orphan Drug Designation for edaravone. The Orphan Drug Act gives special status to a drug that treats a rare disease like ALS and gives incentives for pharmaceutical companies to develop said drug. Mitsubishi Tanabe Pharma is continuing to monitor edaravone's effects in a post-market survey now that it has been approved. This should shed light on whether or not the drug improves survival.

A New Drug Application for edaravone was submitted in the U.S.

A new drug application (NDA) is a means for pharmaceutical companies to formally propose that the FDA approves a new drug for sale and marketing in the U.S. The NDA gives a full picture of the new drug, such as data from the clinic trials and animal studies, how the drug behaves in the body and how it is manufactured, processed and packaged. Mitsubishi Tanabe Pharma submitted edaravone to the FDA to request its approval in the U.S.

What's next?

Edaravone is currently not approved by the FDA for use in the United States. It is not known yet whether the FDA will automatically approve edaravone in the U.S. or whether the FDA will require a phase III study in the U.S.

Questions?

Additional questions about edaravone can be sent to blog@alsa-national.org.

Read full press release from Mitsubishi Tanabe Pharma here.