Belgian biotechnology company Lakefront Biotherapeutics has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to gamgertamig for the treatment of pemphigus.
Gamgertamig, which is being developed by Lakefront in collaboration with Gilead Sciences, was previously granted both Fast Track and Orphan Drug Designation by the US FDA for the treatment of autoimmune haemolytic anaemia (AIHA) and immune thrombocytopenia (ITP).
Ongoing clinical trials of gamgertamig in autoimmune diseases are actively enrolling, with an expansion of the clinical development programme anticipated in 2027.
A Phase 1b study in patients with autoimmune cytopenias, including ITP and AIHA, is currently active in Australia and the US.
A separate Phase 1b study evaluating the drug in patients with seropositive autoimmune diseases, including idiopathic inflammatory myositis, Sjögren’s disease, pemphigus vulgaris (PV) and pemphigus foliaceus (PF), is active in Australia, New Zealand, the Czech Republic and Japan.
Lakefront also expects to initiate proof-of-concept basket studies evaluating gamgertamig in additional autoimmune indications.
Lakefront Chief Medical Officer Eric Hedrick said, “I continue to believe gamgertamig has the potential to represent a transformative medicine in autoimmune diseases.”
Gamgertamig is an investigational BCMAxCD3 bispecific T cell engager being developed for the treatment of autoantibody-driven immune-mediated disease.
Lakefront has in-licensed gamgertamig from Keymed Biosciences, which retains the rights to develop the programme in Greater China.
