The Neuroblastoma Treatment Market is expected to witness substantial growth through 2034. Nearly 90% of neuroblastoma cases are diagnosed in children younger than five years, and close to half of high-risk patients experience disease relapse after achieving initial remission, while around 15% develop refractory disease. Standard treatment approaches comprise surgery, chemotherapy, radiation therapy, and stem cell transplantation. Approved therapies currently available include IWILFIN (USWM), DANYELZA (Y-MAbs), QARZIBA (EUSA Pharma), among others. IWILFIN marked a significant milestone as the first FDA-approved therapy designed to lower relapse risk in both pediatric and adult patients with high-risk neuroblastoma, with additional regulatory filings underway across several countries.
The development pipeline remains robust, with promising candidates such as 64Cu/67Cu SARTATE, idasanutlin, SACT-1, and LEE011. Notable recent developments include Essential Pharma’s acquisition of Renaissance Pharma—bringing the Phase II Hu14.18 immunotherapy into its portfolio—and a manufacturing collaboration with AGC Biologics announced in 2024.
DelveInsight’s report, “Neuroblastoma Market Insight, Epidemiology, and Market Forecast – 2034,” delivers an in-depth evaluation of neuroblastoma, encompassing historical trends and future epidemiological projections, as well as market performance across the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. The study assesses current standards of care, emerging treatment options, drug-wise market share, and market size projections from 2020 to 2034 across the seven major markets. Additionally, it explores the treatment landscape, key growth drivers, challenges, unmet needs, and potential opportunities shaping the future 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
Key Highlights from the Neuroblastoma Market Report
- DelveInsight estimates that the neuroblastoma market will expand at a healthy CAGR through 2034.
- Among the seven major markets, the United States recorded the highest incidence of neuroblastoma cases in 2023.
- Approximately 700–800 new neuroblastoma cases are diagnosed annually in the US.
- Approved treatment options include IWILFIN (USWM), DANYELZA (Y-MAbs Therapeutics), QARZIBA (EUSA Pharma), and others.
- In August 2025, Curant Health announced that Curant Rare became the exclusive pharmacy partner for IWILFIN™, a breakthrough therapy aimed at reducing relapse risk in pediatric and adult patients with high-risk neuroblastoma. From August 2025 onward, patients will transition to Curant Rare to ensure continuous access to treatment and an improved patient and caregiver experience through the IWILFIN Cares® program.
- In July 2025, Norgine revealed that Swissmedic approved IFINWIL® (eflornithine) as a standalone treatment for pediatric patients aged one year and above with high-risk neuroblastoma.
- On May 7, 2025, the FDA cleared an IND application allowing Myrio’s lead candidate, PHOX2B PC-CAR T, to enter clinical trials. Developed in collaboration with a leading children’s hospital in Philadelphia, this CAR-T therapy targets the PHOX2B protein identified as a highly specific marker in neuroblastoma cells under the guidance of Prof. John Maris.
- In September 2024, Senhwa Biosciences announced that silmitasertib (CX-4945) received Rare Pediatric Disease Designation from the US FDA for neuroblastoma treatment.
- In August 2024, RedHill Biopharma reported that opaganib was granted orphan drug designation by the US FDA for neuroblastoma.
- In August 2024, Invenra Inc. disclosed that its bispecific antibody INV724 secured both Rare Pediatric Disease and Orphan Drug Designations from the US FDA for neuroblastoma.
- In April 2024, Essential Pharma finalized the acquisition of Renaissance Pharma, gaining access to its lead immunotherapy candidate Hu14.18K322A (Hu14.18), currently in Phase II development for high-risk neuroblastoma.
- Emerging treatment options include UNITUXIN (dinutuximab), QARZIBA (dinutuximab beta), Omburtamab, SARTATE, and others.
- Prominent companies operating in the neuroblastoma treatment market include United Therapeutics, EUSA Pharma, Y-MAbs Therapeutics, Clarity Pharmaceuticals, and several additional players.
Neuroblastoma Overview
Neuroblastoma is a rare pediatric cancer originating from immature nerve cells and primarily affects infants and young children. The disease most commonly develops in the adrenal glands above the kidneys but may also arise in nerve tissues along the spine, chest, abdomen, or pelvis. While the exact cause is unknown, genetic alterations are believed to contribute. Clinical symptoms depend on tumor location and may involve abdominal swelling, bone pain, fatigue, or unintended weight loss. Diagnosis typically includes imaging studies, tissue biopsy, and bone marrow examination. Treatment strategies vary based on disease stage and risk level and may involve surgery, chemotherapy, radiation therapy, stem cell transplantation, and immunotherapy.
Discover Neuroblastoma market dynamics (drivers, barriers), company strategies, and emerging opportunities across the 7MM – Neuroblastoma Treatment Market
Neuroblastoma Epidemiology
In 2023, the United States accounted for the largest share of neuroblastoma incidence among the seven major markets, with approximately 700–800 new cases each year. The UK reports nearly 100 pediatric cases annually in children aged 0–14 years, while Japan records around 150–200 new cases per year, representing about 8% of all pediatric cancers.
Neuroblastoma Epidemiology Segmentation
The epidemiology section of the report presents both historical data and future forecasts segmented by:
- Total incident neuroblastoma cases
- Age-specific incidence of neuroblastoma
- Risk-group-based incidence of neuroblastoma
- Treatable neuroblastoma patient population
Download the report to understand which factors are driving Neuroblastoma epidemiology trends @ Neuroblastoma Patient Population
Neuroblastoma Drug Uptake and Pipeline Development
• 64Cu/67Cu SARTATE (Clarity Pharmaceuticals):
An advanced radiopharmaceutical targeting SSTR2-positive tumors with enhanced specificity and stability. The therapy is currently being evaluated in Phase I/II trials for pediatric high-risk neuroblastoma and has progressed to Cohort III. It has received FDA Orphan Drug Designation for both 64Cu and 67Cu formulations, along with Rare Pediatric Disease Designation for the 67Cu variant.
• SACT-1 (Aptorum Group):
An oral, repurposed small-molecule therapy designed to promote tumor cell death and inhibit MYCN expression, a common feature in high-risk and relapsed neuroblastoma. Intended for combination use with chemotherapy, Aptorum submitted its Phase Ib/IIa trial protocol to the FDA in June 2023 for relapsed or refractory high-risk neuroblastoma. The drug has also obtained 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 Leading Companies
- Omburtamab – Y-MAbs Therapeutics
- SARTATE – Clarity Pharmaceuticals
- Sacituzumab Tirumotecan (MK-2870) – Merck and Kelun-Biotech
- Eflornithine – K C Pharmaceuticals
Neuroblastoma Market Drivers
- Increasing pediatric cancer incidence: Rising neuroblastoma cases among infants and young children are driving demand for improved therapies.
- Advances in diagnostics: Enhanced imaging and molecular profiling support early diagnosis and personalized treatment.
- Robust research pipeline: Active clinical trials exploring targeted therapies, immunotherapies, and combination regimens are accelerating market growth.
- Support from governments and NGOs: Increased funding and awareness initiatives for rare pediatric cancers promote research and patient access.
Neuroblastoma Market Barriers
- High cost of treatment: Expensive therapies such as immunotherapies and stem cell transplants restrict affordability and access.
- Limited approved options: A shortage of effective therapies persists, especially for relapsed or refractory disease.
- Treatment-related toxicities: Adverse effects from chemotherapy and radiation reduce patient adherence.
- Biological complexity: Genetic and molecular diversity complicates drug development and treatment standardization.
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
- Geographic coverage: 7MM (United States, EU5—Germany, France, Italy, Spain, UK—and Japan)
- Key companies: United Therapeutics, EUSA Pharma, Y-MAbs Therapeutics, Clarity Pharmaceuticals, and others
- Key therapies: UNITUXIN, QARZIBA, Omburtamab, SARTATE, and additional candidates
- Therapeutic assessment: Marketed and emerging neuroblastoma therapies
- Market dynamics: Key drivers and barriers
- Competitive intelligence: SWOT, PESTLE, Porter’s Five Forces, BCG Matrix, and market entry strategies
- Additional insights: Unmet needs, KOL opinions, analyst perspectives, market access, and reimbursement analysis
Table of Contents
- Introduction to the Neuroblastoma Market Report
- Neuroblastoma Executive Summary
- Neuroblastoma SWOT Analysis
- Patient Share Overview (%)
- Market Overview Snapshot
- Disease Background and Overview
- Epidemiology and Patient Population
- Country-Specific Patient Analysis
- Current Treatment Practices
- Unmet Medical Needs
- Emerging Therapies
- Market Outlook
- Country-Wise Market Analysis (2020–2034)
- Market Access and Reimbursement
- Market Drivers
- Market Barriers
- Appendix
- Research Methodology
- DelveInsight Capabilities
- Disclaimer
- About DelveInsight
About DelveInsight
DelveInsight is a prominent healthcare business consulting and market research firm dedicated exclusively to the life sciences sector. The company supports pharmaceutical organizations with end-to-end strategic solutions designed to enhance decision-making and overall performance.
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