Ironwood Pharmaceuticals Announces FDA Orphan Drug Designation for Olinciguat for the Treatment of Sickle Cell Disease

Ironwood Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to olinciguat (IW-1701) for the treatment of patients with sickle cell disease. Olinciguat is an orally administered soluble guanylate cyclase (sGC) stimulator.

“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, M.D., Ph.D., 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. The designation is also an important milestone in Ironwood’s evolution as we advance our pipeline of sGC stimulators focused on the treatment of serious and orphan diseases.”

The FDA’s Office of Orphan Drug Products grants orphan status to drugs intended to treat rare disorders that affect fewer than 200,000 people in the U.S. The designation provides certain benefits to the drug developer, including seven years of market exclusivity upon FDA approval, prescription drug user fee waivers and tax credits for qualified clinical trials.

Ironwood is currently enrolling patients in STRONG-SCD, a multicenter, randomized, double-blind, placebo-controlled, dose-ranging Phase II trial evaluating olinciguat for the potential treatment of sickle cell disease. Ironwood expects to enroll approximately 80 patients into the Phase II trial, which is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of olinciguat in patients with sickle cell disease. Further details about the trial can be found at clinicaltrials.govusing the identifier number NCT03285178.