The experimental drug olinciguat (IW-1701) has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of sickle cell disease. The full article can be found here, at BusinessWire.

Sickle cell disease (SCD) is a group of conditions caused by abnormal production of red blood cells. The red blood cells are created in an unusual ‘sickled’ shape, which causes them to have a shorter lifespan and means that they can become stuck as they move around the bloodstream. It is a serious, lifelong condition that can cause symptoms such as an increased risk of infection, anaemia, and sickle cell crises, which are episodes of pain that can be excruciating. Approximately 100,000 people in the US are thought to be affected by SCD, with some groups, including African Americans, at greater risk.

Olinciguat has been developed by Ironwood Pharmaceuticals as a potential treatment for SCD. It has just been granted Orphan Drug Designation by the FDA.

Orphan Drug Designation is reserved for treatments of rare disorders that affect 200,000 people or fewer in the US. It is intended to speed up the development process, and provide developers with incentives, such as a longer period of marketing exclusivity following approval, fee waivers, and tax credits.

This designation follows encouraging clinical trial evidence about the effects of olinciguat. Olinciguat will be further investigated in a Phase II clinical trial called Strong-SCD, which is currently enrolling patients. It is taking place at multiple centres and will be a randomised, double-blind trial that tests the drug against a placebo. Approximately eighty patients are hoped to take part.

Olinciguat is an experimental drug designed for oral use. It is a soluble guanylate cyclase (sGC) stimulator; sGC is an enzyme involved in vasodilation (blood vessel widening). The sGC is involved in processes that have been linked to SCD, and Ironwood says that it “has the potential to address the underlying causes” of SCD.

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