The status gives incentives for companies to develop new therapies targeting rare and serious diseases, providing marketing exclusivity for seven years and exemption from marketing application fees. The decision will help Revalesio continue the clinical development and evaluation of RNS60.
“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,” Greg Archambeau, president of Revalesio, said in a press release.
While most other biologics and small molecules target a single protein, RNS60 was designed to disrupt inflammatory signals, preventing a series of biochemical events that cause cell damage and death.
“Our treatment impacts the underlying energetic deficits in cells, rather than a single molecular target,” Archambeau said. “Having personally seen the devastating effects of ALS in a Revalesio colleague, we are motivated to find treatments that improve the health and quality of life for patients with ALS.”
ALS, also known as Lou Gehrig’s disease, is a progressive neurological disease that destroys nerve cells and causes disability.
Preclinical results have shown that RNS60 has anti-inflammatory and neuroprotective effects in animal models of ALS, multiple sclerosis, Alzheimer’s disease, and Parkinson’s disease.
RNS60, Revalesio’s lead clinical pharmaceutical candidate, is currently being evaluated in ALS patients in an exploratory, open-label Phase 1 study (NCT02525471) at Massachusetts General Hospital.
All 18 participants will receive intravenous RNS60 (350 ml) once a week. On the remaining six days, patients will inhale 4 ml of the drug. This will be repeated for a total of 24 weeks, but patients will have the opportunity to extend treatment time to a maximum of 48 weeks.
RNS60 will also be tested in ALS patients during a larger, placebo-controlled Phase 2 trial (NCT02988297). The study, to be conducted at 20 clinical centers across Italy and at Massachusetts General Hospital, will evaluate nebulized RNS60, given daily for up to 24 weeks. It is set to enroll 142 patients.