New ALS medication

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A new medication that‘s used to treat people who have amyotrophic lateral sclerosis (ALS) re-energized the ALS research community. Dr. Nicholas Maragakis, who is a co-director of the ALS clinic at Johns Hopkins University, says what was learned from the development of edaravone can be applied to the creation of other ALS medications.

Food and Drug Administration approved the medication in May 2017, which marked the agency’s first approval of a treatment of ALS in 22 years.

Edaravone, which will be marketed as Radicava and was expected to be available in the United States in August 2017, is an intravenous infusion that’s administered by a health-care professional. It has the potential to preserve an ALS patient’s ability to swallow, write, dress and walk, among other physical functions. The medication didn’t receive FDA’s breakthrough designation, because it wasn’t studied in comparison with existing therapies. A head-to-head study isn’t a regulatory requirement for a potential ALS medication.

If taken for a year, edaravone will cost $145,524. MT Pharma America developed the medication and will offer co-pay assistance for insured patients. For a patient who doesn’t have insurance, the pharmaceutical company created a program that provides edaravone at no charge if he/she meets income and other requirements.

“We are confident that our progress will help give hope to people with ALS, and our success could bring renewed commitment by industry to continue studying and developing additional therapies for a disease that deserves a cure,” says Thomas Larson, who is the chief commercial officer at MT Pharma America.

FDA is working with multiple pharmaceutical companies that are working on creating ALS medications.