The U.S. Food and Drug Administration (FDA) has recently granted Orphan Drug Designation to Naproxcinod, a CINOD (Cyclooxygenase-Inhibiting Nitric Oxide-Donating) anti-inflammatory candidate, for the treatment of sickle cell disease (SCD) in the United States (US). The drug is developed by Nicox SA and exclusively licensed to Fera Pharmaceuticals in the US. Naproxcinod is an anti-inflammatory treatment that works by releasing nitric oxide and at the same time suppressing the activity of the enzyme cyclooxygenase, which is responsible for producing pro-inflammatory molecules. The treatment is designed to reduce pain and inflammation, and improve blood flow.
Nicox already completed a broad clinical program for naproxcinod in osteoarthritis, including three phase 3 studies with over 2,700 patients. Fera has conducted pre-clinical work on naproxcinod in models of SCD with promising findings furthering the development of the treatment for vaso-occlusive crises (VOCs), a frequent complication of SCD. More info: Fera press release, Sickle Cell Disease News