FDA grants orphan drug status to Lakefront's gamgertamig for pemphigus

Lakefront Biotherapeutics NV announced on August 24, 2026 that the U.S. Food and Drug Administration has granted Orphan Drug Designation to gamgertamig for the treatment of pemphigus.¹ The drug had already received both Fast Track and Orphan Drug Designation from the FDA for autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP).¹

Gamgertamig is being developed by Lakefront in partnership with Gilead Sciences.¹ The company said its ongoing autoimmune disease trials are currently enrolling, with plans to expand the clinical program in 2027.¹

Two trials are underway. One is a Phase 1b study evaluating gamgertamig in patients with autoimmune cytopenias, a category that includes ITP and AIHA, registered as NCT07083960 and running in Australia and the United States.¹ The second is a Phase 1b trial in seropositive autoimmune conditions, covering idiopathic inflammatory myositis, Sjögren's disease, and both pemphigus vulgaris and pemphigus foliaceus, registered as NCT07229144 and active across Australia, New Zealand, the Czech Republic, and Japan.¹ Lakefront also said it plans to start proof-of-concept basket studies testing gamgertamig in additional autoimmune indications.¹

Chief Medical Officer Eric Hedrick said the company was pleased with the designation and called the drug's potential in autoimmune disease "transformative," according to the release.

The molecule is described as an experimental bispecific T cell engager targeting both BCMA and CD3, aimed at diseases driven by autoantibodies.¹ It is currently in Phase 2 testing and is expected to move into registrational studies in 2027.¹ Lakefront noted the compound is licensed from Keymed Biosciences, which retains rights to develop it in Greater China.¹