Global Von Hippel-Lindau Disease Market - 2024-2031

Overview

The global von Hippel-Lindau disease market reached US$ 293.47 million in 2023 and is expected to reach US$ 501.24 million by 2031 growing with a CAGR of 6.92% during the forecast period 2024-2031.

Von Hippel-Lindau syndrome (VHL) is a rare genetic disorder that leads to the development of tumors and cysts in various parts of the body, including the brain, spinal cord, eyes, inner ear, adrenal glands, pancreas, kidneys, and reproductive system.

While most tumors associated with VHL are benign (non-cancerous), there is a risk of malignant (cancerous) tumors, particularly renal cell carcinoma and pancreatic neuroendocrine tumors. Symptoms typically emerge in young adulthood, although they can manifest at any age. The condition is caused by mutations in the VHL gene, which is crucial for regulating cell growth and division.

Market Dynamics: Drivers

Rising prevalence of VHL-related tumors and hemangioblastomas

The demand for the global von Hippel-Lindau disease market is driven by multiple factors. One of the primary factors is the rising prevalence of VHL-related tumors and hemangioblastomas.

Von Hippel-Lindau's disease leads to the development of tumors in various parts of the body, and many of the clinical trial participants also had other cancer types alongside kidney cancer. As per MD Anderson Cancer Center stats in October 2021, 91% of pancreatic neuroendocrine tumors, which are a rare form of pancreatic cancer, and 30% of hemangioblastomas, which are slow-growing tumors located in the brain or spine, reduced in size by at least 30%.

According to National Cancer Institute data in June 2024, Von Hippel-Lindau (VHL) disease is estimated to range from 1 case per 27,000 to 1 case per 43,000 live births in the general population. Also, various research studies stated that on average, VHL affects approximately 1 in every 35,000 newborns.

Furthermore, from the same source, the prevalence of VHL disease, which refers to the total number of cases in a population at a given time, is estimated to be between 1 in 31,000 to 1 in 91,000 individuals. This means that VHL affects somewhere between 1 and 3 people out of every 100,000 individuals in the general population.

Restraints

Factors such as the high cost of treatment development, the limited availability of treatment options, lack of awareness & diagnosis among individuals, and regulatory challenges are expected to hamper the market.

Segment Analysis

The global von Hippel-Lindau disease market is segmented based on tumor site, treatment, end-user, and region.

The Belzutifan segment accounted for approximately 62.8% of the global von Hippel-Lindau disease market share.

The Belzutifan segment is expected to hold the largest market share over the forecast period. Belzutifan is approved for treating adult patients with von Hippel-Lindau (VHL) disease who need therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET) that do not require immediate surgical intervention.

In addition, Belzutifan is indicated for adult patients with advanced renal cell carcinoma (RCC) after they have received treatment with a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor, as well as a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).

Moreover, key players in the industry product launches & approvals, and key developments help to drive this segment growth of the market. For instance, in August 2021, the U.S. Food and Drug Administration (FDA) approved Belzutifan (WELIREG, Merck), a first-in-class hypoxia-inducible factor (HIF) inhibitor, 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 tumors (pNET), but do not require immediate surgery.

According to the New England Journal of Medicine research article in November 2021, individuals with von Hippel-Lindau (VHL) disease experience a high rate of renal cell carcinoma due to the inactivation of the VHL gene, which leads to the continuous activation of the transcription factor hypoxia-inducible factor 2a (HIF-2a). Belzutifan was linked to primarily grade 1 and 2 adverse events and demonstrated effectiveness in patients with renal cell carcinomas as well as other neoplasms associated with von Hippel-Lindau (VHL) disease.

In addition, key player's ongoing R&D studies would propel this market growth. As per the Ocular Oncology and Pathology research publication in November 2024, Belzutifan demonstrates significant potential in preventing vision-threatening complications in patients with VHL-associated retinal hemangioblastomas (RHs). While the optimal dosing for treating ocular tumors requires additional research, patients receiving treatment for RHs could likely be maintained on a lower daily dose of 80 mg.

However, further studies are needed to address the long-term efficacy of Belzutifan and to determine the best approach for incorporating its use into the treatment and surveillance protocols for patients with VHL-associated RHs. Ongoing research and clinical trials will help refine the dosing and treatment strategies for this patient population.

Geographical Analysis

North America accounted for approximately 44.4% of the global von Hippel-Lindau disease market share.

North America region is expected to hold the largest market share over the forecast period owing to the rising prevalence of VHL-related tumors, raising awareness of cancer, and increasing demand for advanced therapies driving this market growth in the region.

As per ACS publication in March 2021, the VHL tumor suppressor gene is responsible for the persistent expression of angiogenic and growth factors. In the United States, von Hippel-Lindau (VHL) disease occurs in roughly 1 out of every 36,000 newborns. The average age at which VHL is diagnosed is around 26 years old. By the time patients reach 65 years of age, the disease has penetrated and manifested in over 90% of cases.

Moreover, key players in the industry more focus on the treatment for VHL disease research studies, government initiatives, and the rising number of clinical trials that would propel this market growth. For instance, in November 2023, International Kidney Cancer Symposium, Dr. Jaleh Fallah, a medical oncologist at the FDA, reviewed the agency's assessment of data from the phase 2 LITESPARK-004 trial (NCT03401788).

This evaluation was pivotal in the approval of Belzutifan (Welireg) for adult patients with von Hippel-Lindau (VHL) disease who need treatment for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNETs) that do not require immediate surgical intervention.

Market Segmentation

By Tumor Site

• Brain
• Spinal Cord
• Eyes
• Inner Ear
• Reproductive Tract
• Kidney
• Others

By Treatment

• Drug Type
  • HIF-2a Inhibitors
• Belzutifan
  • VEGF Inhibitors
• Bevacizumab
• Ranibizumab
• Surgeries
  • Microsurgical Resection
  • Nephron-Sparing Surgery (NSS)
  • Early Radical Resection
• Others

By End-User

• Hospitals & Specialty Clinics
• Ambulatory Surgical Centers
• Others

By Region

• North America
  • U.S.
  • Canada
  • Mexico
• Europe
  • Germany
  • U.K.
  • France
  • Spain
  • Italy
  • Rest of Europe
• South America
  • Brazil
  • Argentina
  • The rest of South America
• Asia-Pacific
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia-Pacific
• Middle East and Africa

Competitive Landscape

The major global players in the von Hippel-Lindau disease market include Merck & Co., Inc., F. Hoffmann-La Roche Ltd, Genentech, Inc., Amgen Inc., Betta Pharmaceuticals Co., Ltd, Amneal Pharmaceuticals, Inc., Reliance Life Sciences, Lupin, Sandoz, Inc., and Biogen among others.

Key Developments

• In December 2023, Merck announced that the U.S. Food and Drug Administration (FDA) has approved WELIREG, an oral hypoxia-inducible factor-2 alpha (HIF-2a) inhibitor. This medication is intended for adult patients with advanced renal cell carcinoma (RCC) who have previously been treated with a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor, as well as a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).
• In August 2021, Merck acquired the rights to Belzutifan, a hypoxia-inducible factor-2 alpha (HIF-2a) inhibitor, as part of a $1.2 billion deal to purchase the small molecule from Peloton Therapeutics.

Why Purchase the Report?

• To visualize the global von Hippel-Lindau disease market segmentation based on tumor site, treatment, end-user, and region and understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of the von Hippel-Lindau disease market with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping is available in Excel consisting of key products of all the major players.

The global von Hippel-Lindau disease market report would provide approximately 62 tables, 64 figures, and 183 pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies