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Immune-Modulating Drug Approved To Treat Myasthenia Gravis

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A first-in-kind immune-modulating drug that arose from decades of basic research at UT Southwestern Medical Center has received approval from the U.S. Food and Drug Administration as a new treatment for adults with a form of myasthenia gravis. This rare and chronic autoimmune disease is characterized by debilitating and potentially life-threatening muscle weakness.

The new drug, efgartigimod alfa-fcab, is an engineered fragment of a human antibody that binds to a cell surface receptor known as the neonatal Fc receptor, or FcRn. Between 1990 and 2015, former UTSW Professor of Immunology E. Sally Ward, Ph.D., led work that characterized this receptor’s role in regulating the levels and persistence of immunoglobulin G (IgG) antibodies. In 2005, her laboratory described an approach to lower antibody levels by blocking FcRn activity, and subsequently demonstrated preclinical proof-of-concept to treat antibody-mediated autoimmune disease. The global immunology company argenx has licensed exclusive patent rights related to this drug from UTSW.

“The development of this FcRn inhibitor came out of the fundamental work on FcRn biology that my group had worked on during the decades that I was on the UTSW faculty,” said Dr. Ward, now a Professor of Molecular Immunology and Director of Translational Immunology at the University of Southampton in England. “Working out the molecular and cell biological processes involved in FcRn biology and its regulation and transport of antibody molecules was a major focus of our work at UTSW over more than two decades, starting when I was an assistant professor there.”

Clinical trials that led to the recent FDA approval found that 68% percent of patients with anti-acetylcholine receptor antibody positive myasthenia gravis responded to efgartigimod, compared to 30% of those taking a placebo. The company is exploring possible uses for the agent in other conditions mediated by IgG. 

Efgartigimod represents Dr. Ward’s second commercial success based on fundamental research conducted at UTSW. That work also led to technology that can extend the half-life of therapeutic antibodies and currently is used in the FDA-approved drug ravulizumab and two antibody therapies against COVID-19, as well as another antibody in development to treat respiratory syncytial virus (RSV).

It is also the second first-in-kind drug developed from basic research at UTSW to be approved by the FDA in the past year. Less than six months ago, belzutifan, a HIF-2α inhibitor, received approval as a treatment for familial kidney cancer.

“Considered together, these approvals mark UTSW as one of the major sources of breakthrough medicines for previously untreatable diseases. We are indeed a center for biotechnology,” said Michael Brown, M.D., Professor of Molecular Genetics and Internal Medicine and joint recipient of the 1985 Nobel Prize in Physiology or Medicine with research partner UTSW colleague Joseph Goldstein, M.D. Dr. Brown helped recruit Dr. Ward to UTSW in 1990.

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