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FDA Approval Sought for New ALS Drug

by | Jun 28, 2016

Pharmaceutical company Mitsubishi Tanabe Pharma Corporation submitted a formal proposal to the United States Food and Drug Administration for approval of a drug to treat ALS. The drug, Edaravone, gets rid of toxic waste that is a normal by-product of cell function. It is thought that this waste is not as effectively removed in ALS patients, causing damage to motor neurons, which are the cells that die in ALS patients. Edaravone would protect these neurons by helping rid the cells of this waste.

The Edaravone new drug application is supported by clinical research with ALS patients in Japan, and in 2015, Edaravone was approved as a treatment for ALS in Japan and South Korea. After being submitted to the FDA for consideration, it could either be automatically approved for use in the U.S., or it could require a Phase III study conducted in the U.S.

The new drug application gives a full picture of the drug, including data from clinical trials and animal studies, how the drug affects the body, as well as how it is manufactured, processed, and packaged. This submission is Mitsubishi Tanabe Pharma’s first step in accelerating its business development activities in the U.S. The company is also working toward commercializing approved pharmaceutical products in the U.S. and expanding its product line through collaborations with other business partners.

“There is an urgent need for new treatment approaches that may affect the course of this devastating disorder,” said Lucie Bruijn, Ph.D., MBA, Chief Scientist of The ALS Association. “The ALS Association is encouraged by the efforts of Mitsubishi Tanabe Pharma to make this drug available to people living with ALS in the U.S.”

Originally, Edaravone was approved in Japan to treat stroke patients. Studies were conducted in people living with ALS to confirm the safety and effectiveness of Edaravone, and results showed that the drug had the greatest benefit with statistically significant improvement on people who had the disease less than two years. The studies also showed that Edaravone has no benefit on people with more advanced ALS cases.

Edaravone is used as a treatment for ALS in Japan and South Korea through intravenous injections of the drug and in 2015 the FDA and European Commission granted Orphan Drug Designation for Edaravone. The Orphan Drug Act gives special status to drugs that treat rare diseases such as ALS and provides incentives to pharmaceutical companies to develop these drugs. Mitsubishi Tanabe Pharma continues to monitor the effects of Edaravone on ALS patients, which should show whether or not the drug improves chances of survival.

Treeway BV in the Netherlands is currently conducting clinical trials to test an oral version of Edaravone; taking the drug orally would be less invasive and more convenient to treat chronic ALS compared to intravenous injections of the drug. This European study is currently open only to people living in proximity to participating sites.

The FDA will consider whether the drug is safe and effective in its proposed use and whether the benefits of the drug outweigh the risks. The FDA will also review the proposed labeling and the methods used in manufacturing the drug. After the evaluation, the FDA will determine if Edaravone will be approved or if another study is required.

Read full press release from Mitsubishi Tanabe Pharma here.

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