Ouro Medicines announced that gamgertamig (OM336), the company's BCMAxCD3 T cell engager antibody candidate, has been granted US Food and Drug Administration (FDA) Orphan Drug Designation (ODD) for development in the treatment of immune thrombocytopenia (ITP). ITP is an autoimmune disease driven by autoantibodies which cause destruction of platelets, leading to signs and symptoms that include bleeding and fatigue.

"The FDA's decision to grant Orphan Drug Designation to gamgertamig for development in the treatment of ITP means that gamgertamig has now received ODD for two indications in our ongoing Phase 1b autoimmune cytopenias basket study. This second designation underscores the promise for gamgertamig to address unmet needs in ITP, offering an immune reset approach with differentiated properties as a clinical candidate. With dosing now complete for the first cohort in this study and enrollment actively ongoing in the second cohort, we continue to expect results from this study in 2026," said Jaideep Dudani, PhD, CEO, Ouro Medicines.

The US FDA ODD programme is intended to advance therapeutics developed to treat, prevent or diagnose diseases that affect fewer than 200,000 people in the US, and provides benefits including seven years of marketing exclusivity following a potential approval.