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Ironwood's sickle cell disease treatment gets FDA orphan drug designation

Ironwood Pharmaceuticals Inc. said the U.S. Food and Drug Administration granted an orphan drug designation to olinciguat to treat sickle cell disease, an inherited red blood cell disorder.

Sickle cell disease causes red blood cells to deform into a sickle shape and rupture, which creates a shortage of nitric oxide, an important regulator of blood flow. The resulting disruption of blood flow to organs and tissues can lead to fatal conditions including strokes and complications related to the lungs.

Olinciguat is an investigational soluble guanylate cyclase stimulator with a potential to improve NO signaling and improve blood flow.

The Cambridge, Mass.-based biotechnology company is currently enrolling patients to test olinciguat in a phase 2 trial for treating sickle cell disease.

Ironwood is also developing the drug for treating achalasia, a rare disorder that makes it difficult for food and liquid to pass into the stomach.