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Potential treatment targeting nitric oxide levels, olinciguat, named orphan drug by FDA


The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to olinciguat (IW-1701) as a potential treatment of sickle cell disease (SCD), Ironwood Pharmaceuticals announced.

Evidence suggests that the symptoms of sickle cell disease may be associated with nitric oxide deficiency in the blood. Nitric oxide is a free radical produced by several cells and used as a signaling molecule that works to induce cellular events that promote blood flow. Patients with sickle cell anemia have a reduced bioavailability of nitric oxide.

Olinciguat is an oral investigational therapy that stimulates an enzyme called soluble guanylate cyclase (sGC), known to play a key role in the production of nitric oxide.

The stimulation of sGC can restore the bioavailability of nitric oxide in the blood to increase blood flow, which may stop the destruction of blood cells and help address disease symptoms.