Shernan Holtan, MD

FDA Grants Orphan Drug Designation for New Treatment for Life-Threatening Acute GVHD Developed at the University of Minnesota

The FDA has granted Orphan Drug Designation to urinary-derived human chorionic gonadotropin and epidermal growth factor (uhCG/EGF, most commonly available as Pregnyl®) for the treatment of acute graft-versus-host-disease (GVHD). This designation will expedite the development and review of uhCG in treating acute GVHD, a potentially life-threatening complication following a hematopoietic cell transplant.

GVHD can occur after a blood or marrow transplant when the donor immune cells recognize the recipient as foreign and attack certain organs and tissues of the patient, most commonly the skin, gastrointestinal tract, and liver. Patients with severe acute GVHD are at a high risk of death from the damage to their organs and infections due to low immune system function.

More than 13 years of research by Shernan Holtan, MD, Associate Professor of Medicine, Division of Hematology, Oncology, and Transplantation at the University of Minnesota Medical School, and Medical Director, Clinical Trials Office - Cellular, Gene, and Immunotherapy, led to the development of uhCG/EGF as supportive care for severe acute GVHD.

“The goal of our research is to find safe, effective, accessible, and inexpensive supportive care for patients suffering from GVHD,” said Dr. Holtan. “Our research found that giving uhCG in addition to standard treatment may improve outcomes by promoting immune tolerance and tissue repair. We also found that uhCG provides anabolic boost, temporarily increasing testosterone levels, which could have short-term benefits to patients suffering with acute GVHD.”

Dr. Holtan and her team recently published the results of their phase I study in Blood Advances ( The phase II results will be available in 2021.

This development is another first for the University of Minnesota Blood and Marrow Transplantation (BMT) program, which has a remarkable history dating back to 1968. The research was supported by a grant from Regenerative Medicine Minnesota. The team received no pharmaceutical industry support to achieve this designation from the FDA. The Orphan Drug Designation Program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis, or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

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