The Food and Drug Administration (FDA) has granted Orphan Drug Designation to olinciguat (Ironwood Pharmaceuticals) for the treatment of sickle cell disease.

Olinciguat is a soluble guanylate cyclase stimulator currently in Phase 2 development. The Company is enrolling patients in the STRONG-SCD trial which will evaluate the safety and tolerability of 3 dose levels of olinciguat compared with placebo when administered daily for approximately 12 weeks to patients with stable sickle cell disease. 

In non-clinical studies, this investigational agent has been shown to modulate the nitric oxide/soluble guanylate cyclase/cyclic guanosine monophosphate signaling pathway which is believed to be implicated in sickle cell disease.

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“There is an urgent need for new, innovative treatments for patients with sickle cell disease, a debilitating and potentially fatal inherited blood disorder that causes painful crises, organ damage and other serious complications,” said Christopher Wright, MD, PhD, senior vice president of global development and chief development officer. “The orphan drug designation adds momentum to our clinical program investigating olinciguat, which has the potential to improve multiple aspects of sickle cell disease pathophysiology.”

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