DelveInsight’s “Von Hippel-Lindau Disease (VHL) Disease Market Insight, Epidemiology and Market Forecast – 2034” report delivers an in-depth analysis of Von Hippel-Lindau disease epidemiology, market, and clinical development understanding of top oncogenic drivers/biomarkers in Von Hippel-Lindau disease , Addition to this report provides historical and forecasted epidemiology and market data as well as a detailed analysis on the Von Hippel-Lindau disease market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

 

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Key Takeaways from the Von Hippel-Lindau (VHL) Disease Market Report

• Among the 7MM, the United States accounted for the highest number of total prevalent cases of Von Hippel-Lindau disease, i.e. ~9,500 in 2023.
• In EU4 and the UK, Germany accounted for the highest number of total Diagnosed cases of Von Hippel-Lindau disease, whereas Spain accounts for the lowest, in 2023.
• In 2023, in the US, the most prevalent clinical manifestation-specific cases of Von Hippel-Lindau disease in the United States were attributed to retinal hemangioblastomas, making up approximately ~21% of the cases.
• The leading Von Hippel-Lindau (VHL) Disease Companies such as Roche, Exelixis, Bayer, Novartis, and others.
• Promising Von Hippel-Lindau (VHL) Disease Therapies such as Belzutifan, and others.

 

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Von Hippel-Lindau (VHL) Disease Epidemiology Insights in the 7MM

• Total Von Hippel-Lindau Prevalent cases

• Total Von Hippel-Lindau Diagnosed cases

• Von Hippel-Lindau Clinical Manifestation-specific cases

• Total Von Hippel-Lindau treated cases

 

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Von Hippel-Lindau Disease Marketed Drugs

• WELIREG (belzutifan): Merck

WELIREG (belzutifan), developed by Merck, received FDA approval in August 2021. It’s indicated for adult Von Hipple-Lindau patients with renal cell carcinoma, CNS hemangioblastomas, or pancreatic neuroendocrine tumors not immediately needing surgery. Belzutifan inhibits HIF-2α, a key factor in oxygen sensing and gene regulation under hypoxia. By binding to HIF-2α, belzutifan disrupts its interaction with HIF-1β, reducing the expression of genes linked to proliferation, angiogenesis, and tumor growth. WELIREG approval was supported by LITESPARK-004 (NCT03401788), an open-label trial involving 61 Von Hipple-Lindau disease associated RCC patients with a documented Von Hipple-Lindau disease germline alteration and at least one measurable kidney tumor according to RECIST v1.1 criteria.

 

Emerging Von Hippel-Lindau Disease Drugs

• DFF332: Novartis

DFF332 is a small molecule being developed by Novartis as a single agent and in combination in patients with advanced clear cell renal cell carcinoma and other malignancies with HIF stabilizing mutations. The development in combination includes Everolimus (RAD001, an mTOR inhibitor), and also in combination with Spartalizumab (PDR001, an anti-PD1) plus Taminadenant (NIR178, an adenosine A2A receptor antagonist). DFF332 targets a protein called HIF2α; by acting on HIF2α, DFF332 may stop the growth of certain types of cancer. DFF332 is currently being evaluated in a clinical Phase I trial to treat advanced/relapsed ccRCC and other malignancies with HIF2α stabilizing mutations.

 

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Von Hippel-Lindau (VHL) Disease Drugs Market

The existing Von Hippel-Lindau Disease treatment is mainly dominated by Hypoxia-inducible factor-2α (HIF-2α) inhibitor. Belzutifan is an inhibitor of hypoxia-inducible factor 2 alpha (HIF-2α). HIF-2α is a transcription factor that plays a role in oxygen sensing by regulating genes that promote adaptation to hypoxia. Under normal oxygen levels, HIF-2α is targeted for ubiquitin-proteasomal degradation by Von Hipple-Lindau disease protein. Lack of functional Von Hipple-Lindau disease protein results in stabilization and accumulation of HIF-2α. Upon stabilization, HIF-2α translocates into the nucleus and interacts with hypoxia-inducible factor 1 beta (HIF-1) to form a transcriptional complex that induces expression of downstream genes, including genes associated with cellular proliferation, angiogenesis, and tumor growth. Belzutifan binds to HIF-2α, and in conditions of hypoxia or impairment of Von Hipple-Lindau disease protein function, belzutifan blocks the HIF-2α-HIF-1 interaction, leading to reduced transcription and expression of HIF-2α target genes.

 

Von Hippel-Lindau Disease Market Outlook

For patients who have Von Hippel-Lindau disease, the goal of treatment is cancer control, not to cure cancer, and preservation of functional parenchyma to avoid the morbidity associated with renal or adrenal loss. Primary treatment of all Von Hippel-Lindau disease related tumors is local (i.e., surgical resection, RFA, radiotherapy). However, repeated local interventions at multiple sites or repeated recurrences in one area can increase morbidity. Therefore, in individual patients, systemic treatment may be an option.

 

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Scope of the Von Hippel-Lindau (VHL) Disease Market Report

• Coverage- 7MM
• Study Period- 2020-2034
• Von Hippel-Lindau (VHL) Disease Companies- Roche, Exelixis, Bayer, Novartis, and others.
• Von Hippel-Lindau (VHL) Disease Therapies- Belzutifan, and others.
• Von Hippel-Lindau (VHL) Disease Market Dynamics: Von Hippel-Lindau (VHL) Disease Market drivers and Von Hippel-Lindau (VHL) Disease Market Barriers
• Von Hippel-Lindau (VHL) Disease Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
• Von Hippel-Lindau (VHL) Disease Unmet Needs, KOL’s views, Analyst’s views, Von Hippel-Lindau (VHL) Disease Market Access and Reimbursement

 

 

Table of Content

1 Key Insights

2 Report Introduction

3 Executive Summary

4 Key Events

5 Epidemiology and Market Forecast Methodology

6 Von Hippel-Lindau disease Market Overview at a Glance

7 Disease Background and Overview

8 Treatment and Management

9 Epidemiology and Patient Population

10 Patient Journey

11 Marketed Therapies

12 Emerging Therapies

13 Von Hippel–Lindau Disease: 7 Major Market Analysis

14 Unmet Needs

15 SWOT Analysis

16 KOL Views

17 Market Access and Reimbursement

18 Appendix

19 DelveInsight Capabilities

20 Disclaimer

21 About DelveInsight

 

About DelveInsight

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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