Belzutifan, sold under the brand name Welireg, is an
anti-cancer medication used for the treatment of
von Hippel–Lindau disease-associated
renal cell carcinoma.
It is taken
by mouth.
Belzutifan is an
hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor.
The most common side effects include decreased hemoglobin, anemia, fatigue, increased creatinine, headache, dizziness, increased glucose, and nausea.
Belzutifan is the first drug to be awarded an "innovation passport" from the UK
Medicines and Healthcare products Regulatory Agency
The Medicines and Healthcare products Regulatory Agency (MHRA) is an executive agency of the Department of Health and Social Care in the United Kingdom which is responsible for ensuring that medicines and medical devices work and are acceptably ...
(MHRA).
Belzutifan was approved for medical use in the United States in August 2021.
Belzutifan is the first hypoxia-inducible factor-2 alpha inhibitor therapy approved in the US.
The US
Food and Drug Administration (FDA) considers it to be a
first-in-class medication.
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Medical uses
Belzutifan is
indicated
In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...
for treatment of adults with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery.
Belzutifan was also found to be efficacious in an adolescent who had
Pacak–Zhuang syndrome The Pacak-Zhuang syndrome is a recently described disease manifestation in females that includes multiple paragangliomas or pheochromocytomas and somatostatinomas (in some), both neuroendocrine tumors, and secondary polycythemia associated with ...
with polycythemia and paragangliomas.
History
The FDA granted the application for belzutifan
orphan drug
An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.
The assignment of ...
designation.
References
External links
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Breakthrough therapy
Orphan drugs
Benzonitriles
Fluoroarenes
Sulfonates
Indanes
Diphenyl ethers
Nitriles
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