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Ouro Medicines Receives FDA Orphan Drug Designation for Gamgertamig

Ouro Medicines, a poised biotechnology company, has recently announced a significant achievement in its quest to develop immune reset therapeutics: the U.S. FDA has granted Orphan Drug Designation (ODD) for its candidate, Gamgertamig (OM336), to treat immune thrombocytopenia (ITP). This autoimmune condition is characterized by the maladaptive destruction of platelets due to the body’s autoantibodies, leading to serious health issues such as excessive bleeding and fatigue.

This marks the second indication for which Gamgertamig has received ODD, following its earlier designation for autoimmune hemolytic anemia (AIHA). The recognition by the FDA confirms the therapeutic potential of this innovative treatment, which is currently being evaluated in an ongoing Phase 1b global basket study focusing on autoimmune cytopenias, including both ITP and AIHA. Dosing for the first cohort has been successfully completed, while enrollment for the second cohort is actively progressing, signifying a robust commitment towards finding viable solutions for these challenging conditions.

Dr. Jaideep Dudani, CEO of Ouro Medicines, expressed optimism regarding this milestone, highlighting the favorable implications for patients suffering from chronic immune-mediated disorders. He emphasized how this designation is pivotal in addressing unmet needs within the ITP population, noting that Gamgertamig offers a unique immune reset approach that differentiates it from other available clinical options. Results from this ongoing study are anticipated in 2026, providing further insight into the efficacy and safety of Gamgertamig.

The FDA’s Orphan Drug Program aims to facilitate the development of treatments for rare diseases affecting fewer than 200,000 individuals in the United States. The benefits associated with this designation include seven years of exclusive marketing rights post-approval, which can significantly enhance the commercial viability of the treatment.

Overview of Gamgertamig (OM336)

Gamgertamig is an investigational bispecific antibody designed to target BCMA (B-cell maturation antigen) and CD3 in T cells. Its mechanism of action focuses on inducing cellular cytotoxicity against cells that express BCMA, which are implicated in the development of certain immune-mediated diseases through the production of pathogenic autoantibodies. By selectively depleting these harmful cell populations, Gamgertamig aims to provide lasting relief for patients while mitigating the undesirable effects often associated with traditional immunosuppressive therapies.

One of Gamgertamig’s standout features is its 'detuned' CD3-targeting arm, engineered to mitigate the risk of severe immune activation while maintaining therapeutic potency. This design not only enhances the drug's safety profile but may also improve its overall tolerability for patients. Administered via subcutaneous injection, Gamgertamig has been developed with a long half-life, allowing for potentially less frequent dosing schedules, thereby improving patient compliance and quality of life.

Understanding Autoimmune Cytopenias

Autoimmune cytopenias, such as ITP and AIHA, represent a class of diseases characterized by the immune system's inappropriate attack on its own blood cells. ITP affects platelet levels, leading to critical health complications including life-threatening hematological crises. Meanwhile, AIHA results in the destruction of red blood cells, causing debilitating symptoms like chronic fatigue, dizziness, and other severe complications. Both diseases pose significant challenges and necessitate innovative treatment strategies to improve patient outcomes.

Ouro Medicines is dedicated to advancing its clinical programs with a focus on T cell-engaging therapies aimed at promoting immune resets and achieving durable remissions without the need for ongoing immunosuppression. Founded in 2025 in San Francisco by Monograph Capital in collaboration with GSK, the company has garnered interest from notable investors such as TPG and NEA, reflecting strong confidence in its pipeline and mission.

More information about Ouro Medicines and its groundbreaking initiatives can be found on their official website or through their LinkedIn profile. As they continue to innovate within the field of immune-mediated diseases, their commitment to improving patient lives remains steadfast.