Lakefront Biotherapeutics announced on August 24, 2026, that the U.S. FDA granted Orphan Drug Designation to gamgertamig for treating pemphigus, a rare autoimmune blistering disease.
Gamgertamig is being studied in pemphigus vulgaris and pemphigus foliaceus as part of an active Phase 1b study of seropositive autoimmune diseases (NCT07229144).
The drug is described as a BCMA×CD3 T-cell engager intended to reduce antibody-producing plasma and B cells through a short, subcutaneous treatment course.
Gamgertamig previously received FDA Orphan Drug and Fast Track designations for autoimmune hemolytic anemia and immune thrombocytopenia.
The designation does not represent FDA approval or proof of efficacy. It provides development incentives such as potential tax credits, fee exemptions, FDA support, and—if the drug is eventually approved—up to seven years of market exclusivity for the designated indication.
The company expects gamgertamig to enter registrational studies as early as 2027.
Sources:
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