WHA

WELIREG (belzutifan)

Self-Administration - oral

Diagnosis considered for coverage:
  • von Hippel-Lindau (VHL) disease – treatment of adult patients 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.
Coverage Criteria:

For diagnosis of von Hippel-Lindau (VHL) disease:

  • Dose does not exceed 120 mg once daily; AND
  • Patient is 18 years of age or older; AND
  • Prescribed by or in consultation with an oncologist; AND
  • Medical records document patient requires therapy for one of the following VHL-associated tumors:
    • Renal cell carcinoma (RCC)
    • Central nervous system (CMS) hemangioblastoma
    • Pancreatic neuroendocrine tumor (pNET); AND
  • Medical records document the patient does not require immediate surgery.
Reauthorization Criteria:

For diagnosis of von Hippel-Lindau (VHL) disease:

  • Dose does not exceed 120 mg once daily; AND
  • Patient does not show evidence of progressive disease while on therapy.
Coverage Duration:
  • Initial: 1 year
  • Reauthorization: 1 year
Authorization is not covered for the following:
  • The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.
Additional Information:
  • The incidence of VHL syndrome is estimated to be one in 36,000 individuals. This is a rare genetic disease with an estimated incidence of 10,000 people in the U.S. Patients with VHL disease are at risk for benign blood vessel tumors as well as some cancerous ones, including renal cell carcinoma.
  • Exposure to Welireg during pregnancy can cause embryo-fetal harm. Verify pregnancy status prior to the initiation of Welireg. Welireg can render some hormonal contraceptives ineffective.
  • Monitor for anemia before initiation of and periodically throughout treatment with Welireg. Withhold Welireg until hemoglobin ≥9g/dL, then resume at reduced dose or discontinue.
  • VHL disease is different in every patient, and there are no universal treatment guidelines. Treatment for VHL disease also varies according to the location and size of the tumor. Generally, the goal is to provide treatment for tumors before they grow to a size large enough to cause permanent morbidity/mortality. Treatment of most cases usually involves surgery but certain tumors may be treated with high-dose irradiation.
Policy Updates:

02/15/2022 – New policy approved by P&T.

References:
  1. Welireg [Prescribing Information]. Merck & Co, Inc. Whitehouse Station, NJ. August 2021
  2. ClinicalTrials.gov. Web site. https://clinicaltrials.gov. Identifier: NCT03401788. Accessed October 7, 2021.
  3. Food and Drug Administration. Drugs at FDA. https://www.accessdata.fda.gov/scripts/cder/daf/. Accessed September 17, 2021.
  4. Food and Drug Administration. Press Release: FDA approves belzutifan for cancers associated with von Hippel-Lindau disease. August 13, 2021. FDA Web site. FDA approves belzutifan for cancers associated with von Hippel-Lindau disease.  FDA. Accessed September 17, 2021.
  5. Jonasch E, McCutcheon IE, Gombos DS, et al. Pazopanib in patients with von Hippel-Lindau disease: a single-arm, single-centre, phase 2 trial. Lancet Oncol. 2018;19:1351.
  6. Jonasch E, McCutcheon IE, Waguespack SG, et al. Pilot trial of sunitinib therapy in patients with von Hippel-Lindau disease. Ann Oncol. 2011; 22:2661.
  7. National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology – Kidney Cancer v2.2022. NCCN Web site 2021[a]. kidney.pdf (nccn.org). Accessed October 12, 2021.
  8. National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology – Central Nervous System Cancers v2.2021. NCCN Web site 2021[b]. cns.pdf (nccn.org). Accessed October 12, 2021.
  9. National Institutes of Health (NIH). Von Hippel-Lindau disease information page. NIH Web site. Updated August 18, 2021. Von Hippel-Lindau Disease (VHL) Information Page | National Institute of Neurological Disorders and Stroke (nih.gov). Accessed October 11, 2021.
  10. National Organization for Rare Disorders (NORD). Rare Disease Database: Von Hippel-Lindau Disease. Published 2019. NORD Web site. Von Hippel-Lindau Disease - NORD (National Organization for Rare Disorders) (rarediseases.org). Accessed October 11, 2021.
  11. Oken MM, Creech RH, Tormey DC, et al. Toxicity and response criteria of the Eastern Cooperative Oncology Group. Am J Clin Oncol. 1982;5(6):649-655.
  12. Plon SE and Jonasch E. Clinical Features, diagnosis, and management of von Hippel-Lindau disease. UpToDate Web site. Updated October 7, 2021(a). www.uptodate.com. Accessed October 11, 2021.
  13. Plon SE and Jonasch E. Molecular biology and pathogenesis of von Hippel-Lindau disease. UpToDate Web site. Updated October 7, 2021(b). www.uptodate.com. Accessed October 11, 2021.
  14. Stamatakis L, Such B, Singer EA, et al. Phase II trial of vandetanib in von Hippel-Lindau associated renal cell carcinoma. J Clin Oncol. 2013;31(15):4584.
  15. van Leeuwaarde RS, Ahmad S, Links TP, et al. Von Hippel-Lindau Syndrome. 2000 May 17 [Updated 2018 Sep 6]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2021. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1463/. Accessed October 11, 2021.
  16. VHL Alliance. Treatment Considerations. VHL Alliance Web site. Updated May 7, 2019. Treatment Considerations: VHL Alliance. Accessed October 11, 2021.
  17. VHL Alliance. VHLA Suggested Active Surveillance Guidelines. VHL Alliance Web site. Updated October 9, 2020. Active-Surveillance-Guidelines-2020.pdf (vhl.org). Accessed October 11, 2021.

Last review date: February 15, 2022