Rigel’s R289 For MDS Gains Orphan Drug Status By FDA

Rigel Pharmaceuticals, Inc., a company Targeting blood disorders and cancer, announced that the FDA granted an Orphan Drug Designation for its investigational compound, R289, for MDS (myelodysplastic syndromes) treatment. R289 is a dual highly selective IRAK1 and IRAK4 inhibitor evaluated in Phase 1b clinical trial to evaluate its safety, tolerance, pharmacokinetics and preliminary efficacy in patients' lower-risk MDS who did not respond to prior therapies.

Raul Rodriguez, Rigel's president and CEO, commented, "Receiving Orphan Drug designation for R289 supports the development of this therapeutic candidate for the treatment of MDS and highlights the significant unmet medical need for these patients. Orphan Drug and Fast Track designations and encouraging initial data from our ongoing Phase 1b study in patients with lower-risk MDS represent significant milestones in advancing R289 as a potential new treatment option."

The FDA’s Orphan Drug Designation aims to encourage the development of therapies for rare diseases affecting fewer than 200,000 people in the U.S. This designation provides companies with incentives such as tax credits, exemptions from specific FDA fees, and the possibility of seven years of market exclusivity after the drug is approved. Previously, R289 also received Fast Track designation from the FDA for treating patients with transfusion-dependent lower-risk MDS who had undergone prior treatments.