China NMPA Approves Merck’s Welireg to Treat Von Hippel-Lindau Disease-Associated Tumours

China NMPA Approves Merck’s Welireg to Treat Von Hippel-Lindau Disease-Associated Tumours

By, Admin 26 November 2024

China NMPA approves Merck’s Welireg to treat adult patients with certain types of von Hippel-Lindau disease-associated tumours

Overview

Merck, known as MSD outside of the United States and Canada, announced that the National Medical Products Administration (NMPA) in China has approved Welireg (belzutifan), for the 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 tumours (pNET), not requiring immediate surgery.

About the inhibitor: Welireg

  • Welireg is a first-in-class oral hypoxia-inducible factor-2 alpha (HIF-2a) inhibitor and is the first and only approved HIF-2a inhibitor in China. 
  • This approval is based on objective response rate (ORR) and median duration of response (DOR) results from the phase 2 LITESPARK-004 trial and is the 17th approval of Welireg for these patients globally.

Words from the Merck Research Laboratories

  • This approval of Welireg brings the first and only systemic therapy to adult patients in China with certain VHL disease-associated tumours who, to date, have not had access to a non-surgical treatment option to help manage manifestations of VHL disease,” said Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories. 
  • We are committed to bringing innovative treatment options to patients in need around the world and are proud to offer eligible adult patients in China a first-in-class HIF-2a inhibitor as a possible treatment option.”

Welireg Approval in US

  • In August 2021, Welireg was approved in the US for the treatment of adult patients with VHL disease who require therapy for associated RCC, CNS hemangioblastomas or pNET, not requiring immediate surgery. 
  • The efficacy of Welireg was evaluated in LITESPARK-004, an open-label clinical trial in 61 patients with VHL-associated RCC.
  • In the LITESPARK-004 trial, Welireg showed an ORR of 49% (95% CI, 36-62) in patients with VHL-associated RCC (n=30/61); all responses were partial responses (PR). 
  • Median DOR for these patients was not reached, with ongoing responses ranging from 2.8+ to 22.3+ months; among responders, 56% (n=17/30) maintained a response for at least 12 months.

About the LITESPARK-004

  • Patients enrolled in LITESPARK-004 had other VHL-associated tumours, including CNS hemangioblastomas and pNET. 
  • In patients with VHL-associated CNS hemangioblastomas (n=24) in this trial, Welireg showed an ORR of 63% (95% CI, 41-81) (n=15/24), with a complete response (CR) rate of 4% (n=1/24) and a PR rate of 58% (n=14/24). 
  • Median DOR for these patients was not reached, with ongoing responses ranging from 3.7+ to 22.3+ months; among responders, 73% (n=11/15) maintained a response for at least 12 months. 
  • In patients with VHL-associated pNET (n=12) in this trial, Welireg showed an ORR of 83% (95% CI, 52-98) (n=10/12), with a CR rate of 17% (n=2/12) and a PR rate of 67% (n=8/12). 
  • Median DOR for these patients was not reached, with ongoing responses ranging from 10.8+ to 19.4+ months; among responders, 50% (n=5/10) maintained a response for at least 12 months.

Welireg Approval in US: For Advanced RCC

  • Welireg is also approved in the US for the treatment of adult patients with advanced RCC following a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a vascular endothelial growth factor-tyrosine kinase inhibitor (VEGF-TKI), based on results from the phase 3 LITESPARK-005 trial. 
  • Merck is evaluating Welireg in advanced RCC and other tumour types through a broad clinical development programme, including in phase 2 and 3 trials evaluating Welireg as monotherapy and in combination with other medicines.

About LITESPARK-004 Trial

  • LITESPARK-004 is an open-label phase 2 trial (ClinicalTrials.gov, NCT03401788) evaluating Welireg for the treatment of patients with VHL disease who had at least one measurable solid tumour localized to the kidney and who did not require immediate surgery. 
  • The study enrolled 61 patients who received Welireg (120 mg orally once daily) until disease progression or unacceptable toxicity. 
  • The primary endpoint is ORR in VHL disease-associated RCC. Secondary endpoints in RCC tumours include disease control rate, DoR, time to response, progression-free survival, time to surgery and safety. 
  • Additionally, this study evaluated response rates in other common VHL disease-associated tumours including pNET and CNS hemangioblastomas.

Von Hippel-Lindau Disease

  • Von Hippel-Lindau disease is a rare genetic disease that impacts an estimated 200,000 people worldwide. 
  • Patients with VHL disease are at risk for recurrent, benign blood vessel tumours as well as some cancerous ones. 
  • The most commonly occurring tumour is renal cell carcinoma, a form of kidney cancer, which occurs in about 70% of patients with VHL disease.

Welireg (belzutifan) Indications

  • Welireg (belzutifan) is indicated for the 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 tumours (pNET), not requiring immediate surgery.
  • Welireg is indicated for the treatment of adult patients with advanced renal cell carcinoma (RCC) following a programmed death receptor-1 (PD-1) or programmed death ligand 1 (PD-L1) inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).

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