belzutifan

Belzutifan is an oral medication used to treat certain cancers associated with von Hippel-Lindau (VHL) disease. VHL disease is a genetic disorder that can cause tumors to grow in various parts of the body, including the kidneys, brain, spinal cord, and pancreas. Belzutifan is a HIF-2α inhibitor. It works by blocking the activity of hypoxia-inducible factor-2 alpha (HIF-2α), a protein that plays a role in the growth and development of these tumors. By inhibiting HIF-2α, belzutifan can help to slow or stop tumor growth.

The U.S. Food and Drug Administration (FDA) approved belzutifan in August 2021. It is indicated for adult patients with von Hippel-Lindau disease who require therapy for associated renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors, not requiring immediate surgery. Clinical trials have demonstrated its efficacy in reducing tumor size and disease progression in patients with VHL-associated tumors.

Belzutifan is taken orally, usually once a day. Common side effects can include anemia, fatigue, headache, dizziness, nausea, vomiting, and decreased oxygen levels. Patients taking belzutifan require regular monitoring by their healthcare provider to assess treatment response and manage potential side effects. It is an important therapeutic option for individuals affected by VHL disease, offering a targeted treatment approach for these specific types of tumors.