Report: Orphan Drug Appears to Slow ALS Progression

Masitinib, a drug that’s already received “Orphan Drug Designation” from the U.S. Food & Drug Administration (FDA), appeared to slow the progression of amyotrophic lateral sclerosis (ALS) in early results of a double-blind, phase 2/3 controlled trial, Medscape reported on April 7.

The drug’s manufacturer, AB Science, specializes in developing tyrosine kinase inhibitors that it hopes will be able to treat cancers, chronic inflammatory diseases and neurological degenerative disorders.

Masitinib has been available in the United States to treat cancerous tumors in dogs since 2010. The FDA grants Orphan Drug Designations to drugs developed to treat, diagnose or prevent rare diseases. In some cases, the drugs’ manufacturers are not expected to recover the costs of developing and producing the medications due to the relatively low number of patients who will use it.

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Laurie Watanabe is the editor of Mobility Management. She can be reached at

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