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Medications for Von Hippel-Lindau Syndrome

Other names: VHL; Von Hippel-Lindau Disease

Von Hippel-Lindau Syndrome (VHL) is a rare genetic disease associated with tumors in in multiple organs. VHL associated tumors include hemangioblastomas (blood vessel tumors of the brain, spinal cord, and retina) and retinal angiomas (tumors of the retina). People with VHL are also at increased risk of developing renal cell carcinoma, pancreatic neuroendocrine tumors, and pheochromocytoma.

VHL is diagnosed with a genetic test to determine if there is a genetic mutation in the VHL tumor suppressor gene.

When VHL is inactivated, proteins known as hypoxia-inducible factors (including HIF-2α) can accumulate, leading to the growth of both benign and malignant tumors.

Welireg (belzutifan) is the first FDA approved treatment for von Hippel-Lindau Syndrome. Welireg is a hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor that works by reducing transcription and expression of HIF-2α target genes associated with cellular proliferation, angiogenesis and tumor growth.

Drugs used to treat Von Hippel-Lindau Syndrome

The following list of medications are in some way related to or used in the treatment of this condition.

Filter
Drug name Rating Reviews Activity ? Rx/OTC Pregnancy CSA Alcohol
belzutifan Rate Add review
Rx N

Generic name: belzutifan systemic

Brand name:  Welireg

Drug class: miscellaneous antineoplastics

For consumers: dosage, interactions, side effects

For professionals: AHFS DI Monograph

Welireg Rate Add review
Rx N

Generic name: belzutifan systemic

Drug class: miscellaneous antineoplastics

For consumers: dosage, interactions, side effects

For professionals: Prescribing Information

Legend

Rating For ratings, users were asked how effective they found the medicine while considering positive/adverse effects and ease of use (1 = not effective, 10 = most effective).
Activity Activity is based on recent site visitor activity relative to other medications in the list.
Rx Prescription only.
OTC Over-the-counter.
Rx/OTC Prescription or Over-the-counter.
Off-label This medication may not be approved by the FDA for the treatment of this condition.
EUA An Emergency Use Authorization (EUA) allows the FDA to authorize unapproved medical products or unapproved uses of approved medical products to be used in a declared public health emergency when there are no adequate, approved, and available alternatives.
Pregnancy Category
A Adequate and well-controlled studies have failed to demonstrate a risk to the fetus in the first trimester of pregnancy (and there is no evidence of risk in later trimesters).
B Animal reproduction studies have failed to demonstrate a risk to the fetus and there are no adequate and well-controlled studies in pregnant women.
C Animal reproduction studies have shown an adverse effect on the fetus and there are no adequate and well-controlled studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
D There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
X Studies in animals or humans have demonstrated fetal abnormalities and/or there is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience, and the risks involved in use in pregnant women clearly outweigh potential benefits.
N FDA has not classified the drug.
Controlled Substances Act (CSA) Schedule
M The drug has multiple schedules. The schedule may depend on the exact dosage form or strength of the medication.
U CSA Schedule is unknown.
N Is not subject to the Controlled Substances Act.
1 Has a high potential for abuse. Has no currently accepted medical use in treatment in the United States. There is a lack of accepted safety for use under medical supervision.
2 Has a high potential for abuse. Has a currently accepted medical use in treatment in the United States or a currently accepted medical use with severe restrictions. Abuse may lead to severe psychological or physical dependence.
3 Has a potential for abuse less than those in schedules 1 and 2. Has a currently accepted medical use in treatment in the United States. Abuse may lead to moderate or low physical dependence or high psychological dependence.
4 Has a low potential for abuse relative to those in schedule 3. It has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 3.
5 Has a low potential for abuse relative to those in schedule 4. Has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 4.
Alcohol
X Interacts with Alcohol.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.