Lakefront Biotherapeutics NV  announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to gamgertamig for the treatment of pemphigus. Gamgertamig was previously granted both Fast Track and Orphan Drug Designation by the U.S. FDA for the treatment of autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP).

Gamgertamig is being developed by Lakefront in collaboration with Gilead Sciences. Ongoing clinical trials in autoimmune diseases are actively enrolling, and expansion of the clinical trials program is anticipated in 2027.

• Phase 1b study with gamgertamig in patients with autoimmune cytopenias (including ITP and AIHA) (NCT07083960), active in Australia and the United States.
• Phase 1b study with gamgertamig in seropositive autoimmune diseases (including idiopathic inflammatory myositis, Sjögren’s disease, pemphigus vulgaris (PV), and pemphigus foliaceus (PF)) (NCT07229144) active in Australia, New Zealand, Czech Republic, and Japan.
• Expect to initiate proof-of-concept basket studies assessing gamgertamig in additional autoimmune indications.
  
  

“We’re excited by the U.S. FDA’s award of Orphan Drug Designation for gamgertamig for the treatment of pemphigus as we continue to advance it for patients suffering with this debilitating and potentially life-threatening autoantibody-driven disease,” said Eric Hedrick MD, Chief Medical Officer of Lakefront. “I continue to believe gamgertamig has the potential to represent a transformative medicine in autoimmune diseases.”