Remedy Pharmaceuticals, a privately-held, Phase 3-ready pharmaceutical company focused on bringing life saving hospital-based treatment to people affected by central nervous system (CNS) related edema, has announced that the United States Food and Drug Administration ("FDA") has granted orphan drug designation to its lead drug, CIRARA™, for both the treatment of acute subarachnoid hemorrhage and acute spinal cord injury.
Subarachnoid hemorrhage (SAH) is a life-threatening form of a stroke characterized by increased pressure on the brain due to blood and fluid buildup in the subarachnoid space. It is estimated that up to 30,000 people per year suffer from SAH in the United States. A Phase II pilot study evaluating CIRARA™ as a treatment for SAH patients is planned to begin in the second half of this year.
Spinal cord injury (SCI) is a condition resulting from trauma or damage to the spinal cord leading to loss or impaired function causing reduced mobility or sensation. Approximately 12,000 new cases of SCI occur in the United States each year. Remedy Pharmaceuticals initiated enrollment for a Phase II pilot study of CIRARA™ in SCI patients in the last quarter of 2015.
"We are pleased to receive the orphan drug designation for CIRARA™ for both acute subarachnoid hemorrhage and acute spinal cord injury," notes Sven M. Jacobson, Co-Founder and Chief Executive Officer of Remedy Pharmaceuticals. "This represents two key regulatory milestones for the company and takes us a giant step closer to advancing a treatment that we believe holds great promise for patients suffering from these devastating conditions."
The Orphan Drug Designation program offers a distinctive status to investigational drugs and biologics being tested to treat, diagnose or prevent diseases and disorders that affect less than 200,000 people in the U.S. This designation makes the company eligible for a seven year marketing exclusivity period against market competitors. In addition, it provides certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.
CIRARA™ is a high affinity inhibitor of Sur1-Trpm4 channels, which are upregulated in ischemic and traumatic events. CIRARA is designed to close these channels and thus prevent or reduce edema in a variety of CNS-related indications. CIRARA uses the company's proprietary, patented MPD™ technology. CIRARA is an investigational drug and is not approved by FDA.