Remedy Pharma's CIRARA Gets FDA Orphan Drug Status for Large Acute Ischemic Stroke

NEW YORK, Oct. 22, 2024 -- Remedy Pharmaceuticals, a leader in stroke drug development, has announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Division (OOPD) has granted Orphan Drug Designation for their drug CIRARA. This designation is specifically for the treatment of "large territory acute ischemic stroke," which includes large hemispheric infarctions (LHI).

Large hemispheric infarction (LHI) represents a particularly severe form of ischemic stroke, which is caused by the blockage of one of the two significant arteries in the brain: the internal carotid artery or the middle cerebral artery (MCA). This blockage results in the cessation of blood supply to extensive regions of the brain, leading to severe swelling, a high mortality rate, and significant disability.

Sven Jacobson, CEO of Remedy Pharmaceuticals, commented, "The Orphan Drug Designation for CIRARA highlights the uniqueness of large territory ischemic strokes considering their mechanism and consistently poor outcomes, which differ from smaller strokes. This designation will expedite our mission to deliver CIRARA to patients who face the highest risks of severe disability and death due to acute ischemic stroke."

The FDA's Orphan Drug Designation program is designed to grant orphan status to drugs or biologics intended to treat conditions affecting fewer than 200,000 individuals in the U.S. Drugs with this designation may qualify for various benefits, including tax credits for clinical trials, exemptions from prescription drug user fees, and seven years of marketing exclusivity upon FDA approval.

Large Hemispheric Infarction (LHI) is a critical type of ischemic stroke that significantly impacts a substantial part of the brain. This condition arises when a principal cerebral artery, such as the middle cerebral artery (MCA) or the internal carotid artery (ICA), is obstructed. These arteries are essential as they supply oxygenated blood to extensive regions of the brain.

Treatment for LHI generally involves rapid revascularization techniques to restore blood flow. Methods include mechanical thrombectomy or intravenous thrombolysis, which uses clot-dissolving drugs like tissue plasminogen activator (tPA). Despite these treatments, the extensive nature of the infarction and the resulting brain swelling often lead to worse outcomes compared to smaller strokes. Many patients endure long-term disabilities, and in severe cases, the condition can be fatal.

Remedy Pharmaceuticals, based in New York, is a privately held, clinical-stage pharmaceutical company. The company is dedicated to developing and providing life-saving treatments for individuals affected by acute central nervous system (CNS) diseases and injuries. Through their efforts, they aim to address significant unmet medical needs in the field of CNS disorders.