Ouro Medicines announced that gamgertamig (OM336), the company's BCMAxCD3 T cell engager antibody candidate, has been granted 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," said Jaideep Dudani, Ph.D., Chief Executive Officer of Ouro Medicines. "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."
The U.S. FDA ODD program is intended to advance therapeutics developed to treat, prevent or diagnose diseases that affect fewer than 200,000 people in the U.S., and provides benefits including seven years of marketing exclusivity following a potential approval.
The gamgertamig (OM336) basket study in autoimmune cytopenias is an open-label, multi-site, Phase 1b study conducted in the U.S. and Australia evaluating safety, tolerability and pharmacokinetics of gamgertamig in adult participants with active autoimmune cytopenias, specifically relapsed/refractory ITP, AIHA, or both (NCT07083960). Exploratory endpoints include clinical efficacy measures and blood biomarkers. Gamgertamig is administered via subcutaneous injection in ascending dose cohorts, with the primary endpoint evaluated at Week 12.
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox!
Sign up now!