Tacoma-based biomedical company Revalesio recently announced its investigative drug for ALS was granted an Orphan Drug Designation by the Food and Drug Administration.
The Orphan Drug Designation provides incentives for pharmaceutical companies to develop treatments for rare diseases that affect less than 200,000 people in the U.S. The drug, branded RNS60, has been approved for clinical evaluation in the U.S. and Europe and has shown anti-inflammatory and neuroprotective effects in preclinical disease models of ALS and other neurodegenerative diseases.
ALS, also known as Lou Gehrig’s disease, is a fatal degenerative disorder that compromises motor function and leads to paralysis of voluntary muscles. In the U.S., ALS affects about 15,000 people, and only two drugs have been approved for the disease since 1995. However, no treatment has been shown to prevent the disease, or halt or reverse symptoms.
“With orphan status, we have increased access to FDA to help facilitate RNS60’s drug development plan and, hopefully, bring RNS60 to patients who are suffering from ALS,” said Revalesio president Greg Archambeau in a statement.
In partnership with other biomedical research leaders, Revalesio developed the RNS60 drug, and is dedicated to “improving the lives of patients with neurodegenerative diseases.”
The drug is being evaluated in an exploratory study in ALS patients in Massachusetts, and is being used as part of a larger, placebo-controlled, double-blind study in U.S. and global clinical centers.