Neuroblastoma Market Outlook 2034 – Clinical Trials, Market Size, Medication, Prevalence, Companies by DelveInsight

Neuroblastoma Market

Neuroblastoma Market Summary

The Neuroblastoma Treatment Market is projected to grow significantly by 2034. About 90% of cases occur in children under 5, with nearly 50% of high-risk cases relapsing after initial remission and 15% showing treatment resistance (refractory neuroblastoma). Current treatment options include surgery, chemotherapy, radiation, and stem cell transplantation. Approved drugs include IWILFIN (USWM), DANYELZA (Y-mAbs), QARZIBA (EUSA Pharma), among others. IWILFIN became the first FDA-approved therapy to reduce relapse risk in pediatric and adult high-risk neuroblastoma patients, with ongoing regulatory submissions in multiple countries. The pipeline is strong, featuring candidates like 64Cu/67Cu SARTATE, idasanutlin, SACT-1, and LEE011. Recent industry moves include Essential Pharma’s acquisition of Renaissance Pharma (Hu14.18 immunotherapy, Phase II) and a manufacturing partnership with AGC Biologics in 2024.

DelveInsight’s report, “Neuroblastoma Market Insight, Epidemiology, and Market Forecast – 2034,” provides a comprehensive analysis of Neuroblastoma, covering its historical and projected epidemiology, along with market trends across the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. The report examines current treatment practices, emerging therapies, market share of available drugs, and market size forecasts from 2020 to 2034 across the seven major markets. It also highlights the treatment landscape, market drivers and barriers, unmet medical needs, and growth opportunities, offering insights into the future potential of the Neuroblastoma market.

To Know in detail about the Neuroblastoma market outlook, drug uptake, treatment scenario, and epidemiology trends, Click here: Neuroblastoma Market Forecast

Some of the key facts of the Neuroblastoma Market Report:

• Among the 7MM, the US had the highest number of incident cases of neuroblastoma in 2023.
• There are about 700–800 new cases of neuroblastoma each year in the US.
• Several approved drugs for neuroblastoma include IWILFIN (USWM), DANYELZA (Y-mAbs Therapeutics), QARZIBA (EUSA Pharma), and others.
• In August 2025, Curant Health is proud to announce that Curant Rare is now the exclusive pharmacy partner for IWILFIN™, a groundbreaking therapy used to reduce the risk of relapse in adults and children with high-risk neuroblastoma (HRNB). Beginning in August 2025, patients will transition to Curant Rare with one clear goal in mind: to ensure uninterrupted access to care while enhancing the patient and caregiver experience through the IWILFIN Cares® program.
• In July 2025, Norgine, a leading European specialty pharmaceutical company, is pleased to announce that Swissmedic has approved the registration of IFINWIL® (eflornithine) as monotherapy for the treatment of paediatric patients from one year with high-risk neuroblastoma (HRNB)1.
• On 7 May 2025, the FDA approved an IND application enabling Myrio’s lead product (PHOX2B PC-CAR T) to enter human trials. Myrio, in collaboration the leading children’s hospital in Philadelphia have co-developed a Chimeric Antigen Receptor (CAR-T) therapy for the treatment of neuroblastoma. Under the leadership of Prof. John Maris, a functionally relevant and highly specific protein, called PHOX2B, was identified in neuroblastoma cells and the team elucidated that a peptide from PHOX2B could serve as an excellent target for an immunotherapy to treat this devastating disease.
• In September 2024, Senhwa Biosciences, Inc. (TPEx: 6492) announced that its new drug Silmitasertib (CX-4945) was granted a rare pediatric disease designation (RPDD) by US FDA for the treatment of neuroblastoma.
• In August 2024, RedHill Biopharma Ltd. announced that the US FDA granted orphan-drug designation to opaganib for treatment of neuroblastoma.
• In August 2024, Invenra Inc. announced that its bispecific antibody INV724, developed for neuroblastoma, was awarded both Rare Pediatric Disease (RPDD) and Orphan Drug (ODD) Designations by the US FDA.
• In April 2024, Essential Pharma completed the acquisition of Renaissance Pharma, with its lead asset Hu14.18K322A (Hu14.18), an immunotherapy currently in Phase II clinical development for high-risk neuroblastoma (HRNB).
• Emerging therapies include UNITUXIN (dinutuximab), QARZIBA (dinutuximab beta), Omburtamab, SARTATE, and others.
• Key companies in the neuroblastoma treatment market include United Therapeutics, EUSA Pharma, Y-mAbs Therapeutics, Clarity Pharmaceuticals, and many others.

Neuroblastoma Overview

Neuroblastoma is a tumor that develops from neuroblasts (immature nerve tissue) in an infant or child, usually before the age of five. It most often develops in infancy and may be diagnosed in the first month of life. The tumor most often develops in the adrenal gland but may develop in the neck, chest, or spinal cord. It is considered an aggressive tumor because it often spreads to other parts of the body (metastasize). In most cases, it has spread by the time it is diagnosed. A neuroblastoma can cause a variety of signs and symptoms, including a lump where the tumor is growing, bone pain, diarrhea, and various neurological symptoms. The cause of most neuroblastomas is not known. Rarely, a neuroblastoma is caused by an inherited mutation in a gene, such as the ALK gene or PHOX2B gene.

Discover Neuroblastoma market drivers, barriers, company strategies, and emerging opportunities across the 7MM – Neuroblastoma Treatment Market

Neuroblastoma Epidemiology

In 2023, the US recorded the highest number of neuroblastoma incidence cases among the 7MM, with about 700–800 new cases annually. The UK reports around 100 pediatric cases (ages 0–14) each year, while Japan sees 150–200 cases annually, accounting for roughly 8% of pediatric cancers.

Neuroblastoma Epidemiology Segmentation

The Neuroblastoma epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by:

• Total Incident Cases of Neuroblastoma
• Age-specific Incident Cases of Neuroblastoma
• Incident Cases of Neuroblastoma by Risk Groups
• Treatable Cases of Neuroblastoma

Download the report to understand which factors are driving Neuroblastoma epidemiology trends @ Neuroblastoma Patient Population

Neuroblastoma Drugs Uptake and Pipeline Development Activities

• 64Cu/67Cu SARTATE (Clarity Pharmaceuticals): A next-generation radiopharmaceutical targeting SSTR2-positive cancers with high specificity and stability. Currently in Phase I/II trials for pediatric high-risk neuroblastoma (advanced to Cohort III). It has received FDA Orphan Drug Designation (64Cu & 67Cu) and Rare Pediatric Disease Designation (67Cu) for neuroblastoma treatment.
• SACT-1 (Aptorum Group): An orally administered repurposed small molecule designed to induce tumor cell death and suppress MYCN expression, commonly amplified in high-risk/relapsed neuroblastoma. Intended for use in combination with chemotherapy. In June 2023, Aptorum submitted its Phase Ib/IIa trial protocol to the FDA for relapsed/refractory high-risk neuroblastoma. The drug has also been granted FDA Orphan Drug Designation.

Get a comprehensive view of drug uptake trends, clinical progress, regulatory designations, and market opportunities shaping the neuroblastoma market @ Neuroblastoma Medication and FDA Approvals

Neuroblastoma Therapies and Key Companies

• Omburtamab: Y-mabs therapeutics
• SARTATE: Clarity Pharmaceuticals
• Sacituzumab Tirumotecan (Mk-2870): Merck And Kelun-Biotech
• Eflornithine: K C Pharmaceuticals

Neuroblastoma Market Drivers

• Rising incidence in children: Neuroblastoma accounts for ~8% of pediatric cancers, with significant annual cases across the US, UK, and Japan.
• High unmet medical need: Many children with high-risk neuroblastoma relapse (~50%) or show treatment resistance (~15%), creating demand for novel therapies.
• Advancements in treatment options: Approval of drugs like IWILFIN, DANYELZA, and QARZIBA, and pipeline therapies such as 64Cu/67Cu SARTATE, SACT-1, and Hu14.18.
• Regulatory support: FDA Orphan Drug and Rare Pediatric Disease designations encourage drug development.
• Strategic collaborations and acquisitions: Partnerships (e.g., Essential Pharma with AGC Biologics) are strengthening R&D and commercialization efforts.
• Shift toward precision medicine: Development of targeted therapies and immunotherapies addressing genetic and molecular drivers like MYCN amplification.

Neuroblastoma Market Barriers

• Small patient pool: Being a rare pediatric cancer, the limited number of cases reduces overall market size and commercial viability.
• High relapse and refractory rates: Despite new therapies, outcomes remain poor for many high-risk patients.
• High cost of therapies: Advanced biologics and radiopharmaceuticals pose affordability and accessibility challenges.
• Complex clinical trials: Recruiting pediatric patients for trials is difficult, slowing R&D timelines.
• Regulatory hurdles: Pediatric oncology trials face stricter ethical and safety requirements, delaying approvals.
• Limited awareness in low-incidence regions: Diagnosis and timely treatment may be hindered in certain geographies due to lack of specialized expertise.

To learn more about Neuroblastoma companies working in the treatment market, visit @ Neuroblastoma Clinical Trials and Therapeutic Assessment

Scope of the Neuroblastoma Market Report

• Study Period: 2020–2034
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• Key Neuroblastoma Companies: United Therapeutics, EUSA Pharma, Y-mAbs Therapeutics, Clarity Pharmaceuticals, and many others.
• Key Neuroblastoma Therapies: UNITUXIN (dinutuximab), QARZIBA (dinutuximab beta), Omburtamab, SARTATE, and others.
• Neuroblastoma Therapeutic Assessment: Neuroblastoma current marketed and Neuroblastoma emerging therapies
• Neuroblastoma Market Dynamics: Neuroblastoma market drivers and Neuroblastoma market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
• Neuroblastoma Unmet Needs, KOL’s views, Analyst’s views, Neuroblastoma Market Access and Reimbursement

Table of Contents

1. Neuroblastoma Market Report Introduction

2. Executive Summary for Neuroblastoma

3. SWOT analysis of Neuroblastoma

4. Neuroblastoma Patient Share (%) Overview at a Glance

5. Neuroblastoma Market Overview at a Glance

6. Neuroblastoma Disease Background and Overview

7. Neuroblastoma Epidemiology and Patient Population

8. Country-Specific Patient Population of Neuroblastoma

9. Neuroblastoma Current Treatment and Medical Practices

10. Neuroblastoma Unmet Needs

11. Neuroblastoma Emerging Therapies

12. Neuroblastoma Market Outlook

13. Country-Wise Neuroblastoma Market Analysis (2020–2034)

14. Neuroblastoma Market Access and Reimbursement of Therapies

15. Neuroblastoma Market Drivers

16. Neuroblastoma Market Barriers

17. Neuroblastoma Appendix

18. Neuroblastoma Report Methodology

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.

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