Pheochromocytomas and Paragangliomas Market Analysis

DelveInsight's comprehensive "Pheochromocytomas and Paragangliomas Market Insights, Epidemiology, and Market Forecast 2034" analysis delivers extensive understanding of PCPG, historical and projected epidemiology alongside Pheochromocytomas and Paragangliomas therapeutics market trends across the United States, EU4 nations (Germany, France, Italy, and Spain), the United Kingdom, and Japan.

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Major Highlights from Pheochromocytomas/Paragangliomas Market Analysis

• During November 2025, Merck Sharp & Dohme LLC launched a study to assess the efficacy and safety of belzutifan monotherapy in participants diagnosed with advanced pheochromocytoma/paraganglioma (PPGL), pancreatic neuroendocrine tumor (pNET), von Hippel-Lindau (VHL) disease-associated tumors, advanced wt (wild-type) gastrointestinal stromal tumor (wt GIST), or advanced solid tumors with hypoxia inducible factor-2 alpha (HIF-2α) related genetic alterations. The primary objective of the investigation is to evaluate the objective response rate (ORR) of belzutifan per response evaluation criteria in solid tumors version 1.1 (RECIST 1.1) by blinded independent central review (BICR).

• During November 2025, Perspective Therapeutics performed a study representing Phase I/IIa First-in-Human Investigation of [212Pb]VMT-α-NET Targeted Alpha-Particle Therapy for Advanced SSTR2 Positive Neuroendocrine Tumors. This represents a prospective, multi-center, open-label, radioactivity dose-finding/dose expansion investigation of [212Pb]VMT-α-NET in up to 260 adult subjects diagnosed with unresectable or metastatic SSTR2-expressing neuroendocrine tumors (NETs) who have not received prior peptide receptor radionuclide therapy (PRRT).

• The Pheochromocytomas and Paragangliomas market dimension in the 7MM is projected to expand from USD 314 million in 2025 to USD 662 million in 2034.

• The total number of Pheochromocytomas and Paragangliomas incident cases in the 7MM was nearly 4,900 cases in 2024 and is anticipated to increase during the forecasted period.

• During 2024, it has been documented that germline/somatic mutations are present in nearly 75% of all cases in the US.

• The total number of PCPG incident cases in EU4 and the UK was estimated to be nearly 1,800 cases in 2024.

• Among EU4 and the UK, Germany demonstrated the highest number of incident cases of pheochromocytoma and paraganglioma, while Spain recorded the least number of cases, in 2024.

• Among stage-specific cases, the number of localized PCPG cases was higher than that of metastatic cases in Japan.

• Leading Pheochromocytomas/Paragangliomas pharmaceutical organizations include CHIMERIX, OHARA Pharmaceutical, Merck, Novartis, Perspective Therapeutics, among others.

• Notable Pheochromocytomas/Paragangliomas investigational agents include Lanreotide, Axitinib, Sunitinib, Ultratrace Iobenguane (MIBG) I 131, Cabozantinib 40 mg, Belzutifan, melphalan, among others.

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Pheochromocytomas/Paragangliomas Disease Background

Pheochromocytomas and paragangliomas (PPGLs) represent rare neuroendocrine tumors developing from chromaffin cells, with pheochromocytomas originating in the adrenal medulla and paragangliomas developing from extra-adrenal sympathetic or parasympathetic paraganglia. These tumors are marked by excessive or episodic secretion of catecholamines including epinephrine, norepinephrine, and dopamine, resulting in clinical manifestations that commonly encompass persistent or paroxysmal hypertension, severe headaches, palpitations, diaphoresis, anxiety, and tachycardia, although some paragangliomas may be nonfunctional.

Pheochromocytomas/Paragangliomas Epidemiology Classification in the 7MM

• Pheochromocytomas and paragangliomas Incident Cases

• Pheochromocytomas and paragangliomas Occurrence or Absence of mutation

• Pheochromocytomas and paragangliomas Age-specific Cases

• Pheochromocytomas and paragangliomas Stage-specific Cases

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Pheochromocytomas and Paragangliomas Commercialized Therapeutic Agents

DEMSER Capsule 250 mg (metyrosine): Bausch Health and Ono Pharmaceutical

DEMSER represents a promising drug demonstrating efficacy in the improvement of symptoms in patients who are not able to sufficiently control symptoms with sympatholytic drugs. DEMSER is indicated in the management of pheochromocytoma patients for preoperative preparation of patients for surgery, management of patients when surgery is contraindicated, and chronic treatment of malignant pheochromocytoma patients. DEMSER (ONO-5371) was authorized and launched in the United States in 1979 for managing pheochromocytoma. During January 2019, Ono Pharmaceutical received manufacturing and marketing approval in Japan for DEMSER capsule for improvement of the status of catecholamine excess secretion in pheochromocytoma patients. During July 2020, the US FDA authorized Amneal Pharmaceuticals' generic version of DEMSER (metyrosine) oral capsules. Amneal's generic represents the first for DEMSER to be authorized in the United States.

Pheochromocytomas and Paragangliomas Investigational Therapeutic Agents

ONC201: Chimerix

ONC201, also designated as dordaviprone, represents a first-in-class small-molecule imipridone that selectively binds to the G-protein coupled dopamine receptor D2 (DRD2) and the mitochondrial protease ClpP. ONC201 has been evaluated in an open-label Phase II investigator-initiated investigation that treated 30 patients at the Cleveland Clinic diagnosed with rare neuroendocrine tumors. Paraganglioma patients were enrolled in two cohorts initiating ONC201 either once or twice weekly. ONC201 interim efficacy outcomes in dopamine-secreting tumors outside the brain demonstrated 50% ORR in paraganglioma. Investigator-assessed data from this investigation were presented at the annual meeting of the American Society of Clinical Oncology (ASCO) in 2021 and published in the journal Clinical Cancer Research in 2022.

WELIREG (belzutifan/MK-6482): Merck

WELIREG represents a novel, potent and selective inhibitor of HIF-2a. WELIREG is indicated for von Hippel-Lindau (VHL) disease who require treatment for a type of kidney cancer called Renal Cell Carcinoma (RCC), tumors in the brain and spinal cord called Central Nervous System (CNS) hemangioblastomas, or a type of pancreatic cancer called pancreatic Neuroendocrine Tumors (pNET), that do not require surgery immediately, and kidney cancer that has spread (advanced RCC) following treatment with a PD-1 or PD-L1 and VEGF cancer medicines. It is currently under evaluation in Phase II for managing advanced PCPG, with a target action date of May 26, 2025, under the Prescription Drug User Fee Act (PDUFA).

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Pheochromocytomas and Paragangliomas Pharmaceutical Organizations

CHIMERIX, OHARA Pharmaceutical, Merck, Novartis, Perspective Therapeutics, among others.

Coverage of the Pheochromocytomas and Paragangliomas Market Analysis

• Coverage: 7MM

• Analysis Period: 2020-2034

• Pheochromocytomas/Paragangliomas Pharmaceutical Organizations: CHIMERIX, OHARA Pharmaceutical, Merck, Novartis, Perspective Therapeutics, among others.

• Pheochromocytomas/Paragangliomas Therapeutic Agents: Lanreotide, Axitinib, Sunitinib, Ultratrace Iobenguane (MIBG) I 131, Cabozantinib 40 mg, Belzutifan, melphalan, among others.

• Pheochromocytomas/Paragangliomas Therapeutic Evaluation: Pheochromocytomas/Paragangliomas current commercialized and Pheochromocytomas/Paragangliomas investigational therapies

• Pheochromocytomas/Paragangliomas Market Dynamics: Pheochromocytomas/Paragangliomas market growth factors and Pheochromocytomas/Paragangliomas market obstacles

• Pheochromocytomas/Paragangliomas Competitive Intelligence Evaluation: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry methodologies

• Pheochromocytomas/Paragangliomas Unmet Requirements, KOL perspectives, Analyst perspectives, Pheochromocytomas/Paragangliomas Market Access and Reimbursement

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Report Structure

1. KEY INSIGHTS

2. REPORT INTRODUCTION

3. EXECUTIVE SUMMARY

4. PCPG MARKET OVERVIEW AT A GLANCE

5. KEY EVENTS

6. EPIDEMIOLOGY AND MARKET METHODOLOGY

7. PCPG BACKGROUND AND OVERVIEW

8. PCPG TREATMENT AND MANAGEMENT

9. PCPG EPIDEMIOLOGY AND PATIENT POPULATION

10. PCPG PATIENT JOURNEY

11. PCPG MARKETED DRUGS

12. PCPG DISCONTINUED PRODUCT

13. PCPG EMERGING DRUGS

14. PCPG: SEVEN MAJOR MARKET ANALYSIS

15. PCPG UNMET NEEDS

16. SWOT ANALYSIS

17. KOL VIEWS

18. MARKET ACCESS AND REIMBURSEMENT

19. APPENDIX

20. DELVEINSIGHT CAPABILITIES

21. DISCLAIMER

22. ABOUT DELVEINSIGHT

About DelveInsight

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