MECHELEN, Belgium — August 24, 2026
Lakefront Biotherapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to gamgertamig for the treatment of pemphigus, expanding the regulatory recognition of the investigational autoimmune therapy. The designation adds pemphigus to gamgertamig’s existing U.S. FDA Orphan Drug and Fast Track designations for autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). Developed in collaboration with Gilead Sciences, gamgertamig is being evaluated as a potential treatment for autoantibody-driven immune-mediated diseases, with ongoing clinical studies enrolling patients across multiple autoimmune indications.
FDA Designation Expands Gamgertamig’s Autoimmune Development Program
The FDA’s orphan designation provides an important regulatory milestone for gamgertamig in pemphigus, a group of autoimmune diseases characterized by pathogenic autoantibodies that can cause painful blistering of the skin and mucous membranes. The program is already being investigated in a Phase 1b study in patients with autoimmune cytopenias, including ITP and AIHA, under clinical trial NCT07083960, which is active in Australia and the United States. A second Phase 1b study, NCT07229144, is evaluating gamgertamig in seropositive autoimmune diseases, including pemphigus vulgaris, pemphigus foliaceus, idiopathic inflammatory myositis and Sjögren’s disease. The company expects to expand the clinical development program in 2027 with additional proof-of-concept basket studies across autoimmune indications.
BCMAxCD3 Approach Targets Autoantibody-Driven Disease
Gamgertamig is an investigational BCMAxCD3 bispecific T-cell engager designed to target immune mechanisms involved in autoantibody-driven disease. The therapeutic approach is intended to engage T cells through CD3 while targeting BCMA-expressing plasma cells, potentially addressing a source of pathogenic antibody production. Lakefront is developing gamgertamig across multiple autoimmune diseases rather than limiting the program to a single indication, creating an opportunity to evaluate the same therapeutic platform across diseases with shared autoantibody biology. The company describes gamgertamig as having potential to become a first-in-class and best-in-class autoimmune therapy, although its clinical efficacy and long-term safety remain to be established through ongoing and future trials.
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Pemphigus Adds Another Clinical Opportunity for Gamgertamig
The new FDA designation strengthens the broader development strategy for Lakefront Biotherapeutics, particularly as the company builds its immunology and inflammation pipeline around gamgertamig. The program is being advanced in collaboration with Gilead Sciences, while development rights for Greater China are held by Keymed Biosciences, from which Lakefront in-licensed the program. With studies already active across autoimmune cytopenias and seropositive autoimmune diseases, the addition of pemphigus provides another clinical setting in which to evaluate the potential of the BCMAxCD3 mechanism. Lakefront expects further expansion of the program in 2027, including proof-of-concept basket studies in additional autoimmune indications. The FDA’s orphan designation therefore represents a regulatory development milestone, while the key upcoming catalysts will be clinical data demonstrating whether gamgertamig can deliver meaningful disease control with an acceptable safety profile.
Source:Lakefront Biotherapeutics,press relese
