uhCG plus EGF granted orphan drug designation for the treatment of acute graft-versus-host-disease

On August 18, 2020, urinary-derived human chorionic gonadotropin (uhCG)/epidermal growth factor (EGF) was granted orphan drug designation by the U.S. Food and Drug administration (FDA) for the treatment of acute graft-versus-host-disease (aGvHD).¹ uhCG holds immunomodulatory properties and EGF acts synergistically to support epithelial repair—forming the rationale behind the treatment approach.

The decision was largely based on the results from a phase I study (NCT02525029) in which 26 participants received subcutaneous injections of uhCG/EGF plus conventional immunosuppressive therapy (frontline therapy for high-risk GvHD, n = 13; second-line therapy, n = 13). uhCG/EGF was well tolerated and no dose-limiting toxicities were observed. In terms of efficacy, a complete response at Day 28 was observed in 62% of patients in the high-risk group and 54% of patients receiving second-line treatment. Following 1 week of treatment with uhCG/EGF, the T regulatory cells/conventional T cells ratio increased by more than twofold, alluding to immune modulation. The development of uhCG/EGF for aGvHD is an academic effort and has been made possible through the University of Minnesota Blood and Marrow Transplantation (BMT) program.

Results from the aforementioned phase I study, as presented at the 60th American Society of Hematology (ASH) Annual Meeting, 2018, can be found here.