Pediatric Relapsed-Refractory Neuroblastoma Market

Key Highlights

• Approximately half of the patients with neuroblastoma are diagnosed with a clinically aggressive, high-risk form of disease. Despite advances in the first-line treatment of high-risk neuroblastoma, 10–20% of patients do not respond to treatment, and 50–60% relapse. 
• Relapsed/refractory neuroblastoma has a poor prognosis and was previously considered fatal. 
  The available treatments for pediatric relapsed/refractory neuroblastoma include surgery, chemotherapy, radiation therapy, and stem cell transplantation.
• Several approved drugs for pediatric relapsed/refractory neuroblastoma include IWILFIN (USWM and Norgine), DANYELZA (Y-mAbs Therapeutics), QARZIBA (EUSA Pharma), and others.
• In April 2024, Norgine announced its first marketing authorization application submissions on 10 April 2024, seeking approval for IWILFIN (eflornithine) in high-risk neuroblastoma, via Project Orbis in Australia, Switzerland, and the UK. 
• In January 2025, Norgine announced that it completed its marketing authorisation application filing to European Medicines Agency (EMA) for eflornithine in high-risk neuroblastoma. 
• The pipeline consists of products such as 64Cu SARTATE/ 67Cu SARTATE (Clarity Pharmaceuticals), TECELRA (afamitresgene autoleucel) (Adaptimmune), PEEL-224 (Peel Therapeutics), and others. 
• Currently, the emerging landscape of pediatric relapsed/refractory neuroblastoma is not very robust and holds an unmet need in terms of conducting better trials, due to many trial failures observed in previous years. With growing awareness and many companies in the early Phases, the landscape of the indication is expected to undergo vast changes.

DelveInsight's “Pediatric Relapsed/Refractory Neuroblastoma– Market Insight, Epidemiology and Market Forecast – 2034” report delivers an in-depth analysis of pediatric relapsed/refractory neuroblastoma epidemiology, market, and clinical development in pediatric relapsed/refractory neuroblastoma. In addition to this, the report provides historical and forecasted epidemiology and market data as well as a detailed analysis of the pediatric relapsed/refractory neuroblastoma market trends in the United States, EU4 (Germany, France, Italy, and Spain ), the United Kingdom, and Japan.

The pediatric relapsed/refractory neuroblastoma market report provides real-world prescription pattern analysis, emerging drugs assessment, market share, and uptake/adoption pattern of individual therapies, as well as historical and forecasted pediatric relapsed/refractory neuroblastoma market size from 2020 to 2034 in 7MM. The report also covers current pediatric relapsed/refractory neuroblastoma treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s underlying potential.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Pediatric Relapsed/Refractory Neuroblastoma Understanding and Treatment Algorithm

Pediatric Relapsed/Refractory Neuroblastoma Overview 

Neuroblastoma is a tumor that develops from neuroblasts (immature nerve tissue) in an infant or child (below the age of 18; pediatric), 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. 

Research has led to great strides in neuroblastoma survival. However, roughly 10% to 15% of children with high-risk neuroblastoma do not respond to initial treatment (refractory). In addition, about 40% to 50% of children with neuroblastoma have cancer that returns after treatment (relapse).

Pediatric Relapsed/Refractory Neuroblastoma Diagnosis

The diagnosis is through a comprehensive approach, including physical exams, blood tests, imaging (MRI, CT scans), and biopsy confirmation. Two staging systems are used: the Neuroblastoma Risk Group Staging System (INRGSS), which assesses stage using pre-treatment imaging (L1, L2, M, MS), and the International Neuroblastoma Staging System (INSS), relying on surgical findings (Stages I-IV, 4S). Risk stratification under INRG considers clinical and genetic factors (e.g., MYCN status, chromosome 11q, tumor ploidy) to classify tumors into very low, low, intermediate, or high-risk groups.

Further details related to country-based variations in diagnosis are provided in the report

Pediatric Relapsed/Refractory Neuroblastoma Treatment         

Treatment strategies are tailored to the tumor's size, location, and the child's age. Initial treatment often begins with surgery, followed by chemotherapy, radiation therapy, or stem cell transplantation in severe cases. Some neuroblastomas may resolve spontaneously without treatment. Recurrent neuroblastoma, if it occurs, is managed based on prior treatment history, recurrence site, and initial risk group.

Pediatric Relapsed/Refractory Neuroblastoma Epidemiology

The pediatric relapsed/refractory neuroblastoma epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented as total incident cases of neuroblastoma, total incident cases of pediatric relapsed/refractory neuroblastoma, incident cases of pediatric relapsed/refractory neuroblastoma by risk groups, and treatable cases of pediatric relapsed/refractory neuroblastoma in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), and the United Kingdom, and Japan from 2020 to 2034. 

• Neuroblastoma is the most frequent extracranial solid tumor in children (10% of cancers), with an estimated incidence of 1/8000–10,000 in the North America and Europe.
• Approximately 50% of children with high-risk neuroblastoma experience initial remission followed by cancer relapse. Another 15% of children with high-risk neuroblastoma would not respond to initial treatment. These children are said to have refractory neuroblastoma.
• The incidence of neuroblastoma is approximately one case per 70,000 live newborns, which in Spain amounts to some 100 cases a year. The disease behaves in many different ways: it can be benign and require no treatment (50%-60% of cases) or be aggressive, requiring multimodal treatment (45%-55%).
• Spain reports 80–90 cases of neuroblastoma annually in children aged 0–14, including approximately 45 high-risk neuroblastoma and 20 relapse/progressive cases per year.
• Around 100 children between the ages of 0 and 14 years are diagnosed with neuroblastoma each year in the UK.
• Neuroblastoma occupies nearly 8% of pediatric malignancies and occurs in ~150–200 children each year in Japan. 

Pediatric Relapsed/Refractory Neuroblastoma Drug Chapters

The drug chapter segment of the pediatric relapsed/refractory neuroblastoma report encloses a detailed analysis of pediatric relapsed/refractory neuroblastoma marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also deep dives into pediatric relapsed/refractory neuroblastoma's pivotal clinical trial details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations. 

Marketed Drugs

IWILFIN (eflornithine): USWM (US WorldMeds) and Norgine

IWILFIN is a prescription drug used to reduce the risk of relapse in adults and children with high-risk neuroblastoma that has gotten better after treatment with certain prior therapies. Eflornithine acts as an irreversible inhibitor of the enzyme Ornithine Decarboxylase (ODC). ODC is the first rate-limiting enzyme that plays a vital role in polyamine synthesis and is a transcriptional target of the MYCN gene. Polyamines are molecules involved in cell proliferation and endurance and are crucial for neoplastic transformation. Inhibition of polyamine synthesis by eflornithine restores the LIN28/Let-7 metabolic pathway balance that regulates cancer stem cells and glycolytic metabolism, by reducing expression of the oncogenic drivers MYCN and LIN28B in MYCN-amplified neuroblastoma, thereby suppressing tumor growth and formation. 

In July 2021, US WorldMeds and Norgine announced an exclusive licensing agreement by which Norgine was registered to commercialise eflornithine in Europe.

In December 2023, the FDA approved IWILFIN to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-disialoganglioside 2 (GD2) immunotherapy. The approval was based on efficacy, evaluated in an externally controlled trial comparing outcomes from Study IIIb (investigational arm) and Study ANBL0032. In January 2025, a marketing authorization application was submitted to the European Medicines Agency (EMA) seeking the approval of IWILFIN for the treatment of patients with high-risk neuroblastoma.

DANYELZA (naxitamab): Y-mAbs Therapeutics

DANYELZA is a GD2-binding monoclonal antibody indicated, in specific combination with Granulocyte-Macrophage Colony-Stimulating Factor (GMCSF), for the treatment of pediatric patients one year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. It binds to the glycolipid GD2. GD2 is a disialoganglioside that is overexpressed on neuroblastoma cells and other cells of neuroectodermal origin, including the central nervous system and peripheral nerves. In vitro, naxitamab-gqgk was able to bind to cell surface GD2 and induce complement-dependent cytotoxicity (CDC) and antibody-dependent cell-mediated cytotoxicity (ADCC).

In November 2020, the FDA approved DANYELZA for pediatric patients of one year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow demonstrating a partial response, minor response, or stable disease to prior therapy. This approval was based on Study 201 and Study 12-230.

In November 2024, Y-mAbs Therapeutics announced that they have entered into an exclusive license and distribution agreement with Nobelpharma for the development and commercialization in Japan of DANYELZA for the treatment of patients with relapsed/refractory high-risk neuroblastoma and, upon agreement by the parties, potentially relapsed osteosarcoma. Under the terms of the agreement, Nobelpharma will employ its regulatory, marketing, sales and access expertise to carry out development work and to submit DANYELZA for approval by Japanese regulatory authorities, and to market, sell, and distribute DANYELZA in Japan, if approved.

Note: Detailed current therapies assessment will be provided in the full report of pediatric relapsed/refractory neuroblastoma.

Emerging Drugs

64Cu SARTATE/ 67Cu SARTATE: Clarity Pharmaceuticals

SARTATE is a next-generation, highly targeted theranostic radiopharmaceutical with increased specificity and in-vivo stability, being developed for diagnosing, staging, and subsequently treating cancers that express Somatostatin Receptor 2 (SSTR2). 

Currently, Clarity Pharmaceuticals is conducting the Phase I/II trial to evaluate the safety and efficacy of 67Cu-SARTATE in pediatric patients with high-risk neuroblastoma. The trial has been advanced to Cohort III. 67Cu SARTATE and 64Cu SARTATE have been granted FDA Orphan Drug Designation (ODD) status for the treatment and clinical management of neuroblastoma. The US FDA has also granted Rare Paediatric Disease Designation (RPDD) to 67Cu-SARTATE to treat neuroblastomas. 

PEEL-224: Peel Therapeutics

PEEL-224 is a nanocarrier linked to four small molecules developed for the treatment of cancer. The active molecule of PEEL-224 derives from camptothecin, a naturally occurring compound found in the Chinese Happy Tree, thought to have evolved as a plant defense mechanism. The robust preclinical data is extremely promising, showing that PEEL-224 leads to a sustained complete response in the most aggressive solid tumors in some cases for more than six months.

Currently, the PEEL-224 is being evaluated for the Phase I/II for the solid tumors, including neuroblastoma cohort: Age greater than or equal to 1 year and less than or equal to 30 years.

Note: Detailed emerging therapies assessment will be provided in the final report.

Drug Class Insights

Targeted therapies for mutations, such as DANYELZA, UNITUXIN, IWILFIN, and others, mainly dominate the existing treatment. IWILFIN is associated with the inhibition of ODC. Other emerging drugs include SSTR2 inhibitors, and others which will be a part of the rising market in the upcoming years. GD2 inhibitors have expanded treatment options for pediatric relapsed/refractory neuroblastoma, receiving approvals since 2015.

GD2 inhibitors

GD2 expression levels are associated with tumor cell susceptibility to anti-GD2 antibody cytotoxicity. Serum levels of GD2 are significantly higher in children with high-risk neuroblastoma compared to controls, suggesting a potential role of GD2 as a tumor biomarker for diagnosis or prognosis. There are several marketed drugs for neuroblastoma, like DANYELZA, UNITUXIN, that have shown efficacy by inhibiting GD2. 

In the emerging pipeline, some of the major classes observed are SSTR2 inhibitor (64Cu SARTATE/ 67Cu SARTATE) and PEGylated TOP1 inhibitor (PEEL-224).

Pediatric Relapsed/Refractory Neuroblastoma Market Outlook

The market outlook for neuroblastoma treatment is promising, driven by advancements in targeted therapies and immunotherapies. Immunotherapy directed against the GD2 antigen is becoming part of the standard treatment protocols for patients who are not in remission despite prior frontline therapies, including chemotherapeutic agents. Studies are also investigating the potential of these agents to consolidate treatment response to chemotherapy. Most research studies about high-risk neuroblastoma focus on finding the best combinations of chemotherapy drugs, stem cell transplant regimens, immunotherapies, and other new treatments to try to cure more children. Current studies of low- and intermediate-risk neuroblastoma are trying to figure out if children can get less treatment and still do as well. Current real-time treatments involve surgery followed by chemotherapies, radiation, and retinoid therapies (Isotretinoin). Currently, the approved drugs for pediatric relapsed/refractory neuroblastoma include IWILFIN, DANYELZA, etc.

Key players, such as Clarity Pharmaceuticals, Adaptimmune, Peel Therapeutics, and others, are evaluating their candidates in different stages of clinical development. They aim to investigate their products for the treatment of pediatric relapsed/refractory neuroblastoma.

Key Findings

• In May 2025, Y-mAbs Therapeutics announced that DANYELZA is recommended by the NCCN (National Comprehensive Cancer Network) Clinical Practice Guidelines in Oncology (NCCN guidelines) as a NCCN Category 2A treatment option for high-risk neuroblastoma.
• In January 2025, USWM submitted a marketing authorization application to the EMA seeking the approval of IWILFIN for the treatment of patients with high-risk neuroblastoma.

Pediatric Relapsed/Refractory Neuroblastoma Drug Uptake

When comparing IWILFIN, DANYELZA, and UNITUXIN, DANYELZA has the highest market uptake. DANYELZA is the only FDA-approved option for high-risk neuroblastoma in the bone and/or bone marrow, specifically for relapsed or refractory disease. It targets a glycolipid called GD2, which is prevalent in neuroblastoma cells and contributes to their growth and survival.

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024–2034, which depends on the competitive landscape, safety, and efficacy data, along with the order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake. 

Further detailed analysis of emerging therapies' drug uptake in the report…

Pediatric Relapsed/Refractory Neuroblastoma Activities

The report provides insights into different therapeutic candidates in the Phase III and Phase II stages. It also analyzes key players involved in developing targeted therapeutics. 

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Pediatric Relapsed/Refractory Neuroblastoma therapies. 

KOL Views

To keep up with the real-world scenario in current and emerging market trends, we take opinions from Key Industry leaders working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on the evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility, including Medical/scientific writers, Professors, and Others.

DelveInsight’s analysts connected with 20+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as UT Health San Antonio MD Anderson Cancer Center, etc., were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or pediatric relapsed/refractory neuroblastoma market trends. 

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. 

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

In efficacy, the trial’s primary and secondary outcome measures are evaluated; for instance, in event-free survival, one of the most important primary outcome measures is event-free survival and overall survival. 

Further, the therapies’ safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided. 

Market Access and Reimbursement

Reimbursement may be referred to as the negotiation of a price between a manufacturer and payer that allows the manufacturer access to the market. It is provided to reduce the high costs and make the essential drugs affordable. Health technology assessment (HTA) plays an important role in reimbursement decision-making and recommending the use of a drug. These recommendations vary widely throughout the seven major markets, even for the same drug. In the US healthcare system, both Public and Private health insurance coverage are included. Also, Medicare and Medicaid are the largest government-funded programs in the US. The major healthcare programs including Medicare, Medicaid, Health Insurance Program (CHIP), and the state and federal health insurance marketplaces are overseen by the Centers for Medicare & Medicaid Services (CMS). Other than these, Pharmacy Benefit Managers (PBMs) and third-party organizations that provide services and educational programs to aid patients are also present.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report

• The report covers a segment of key events, an executive summary, descriptive overview of pediatric relapsed/refractory neuroblastoma, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of the diagnosis rate, and disease progression along treatment guidelines.
• Additionally, an all-inclusive account of both the current and emerging therapies, along with the elaborative profiles of late-stage and prominent therapies, will have an impact on the current treatment landscape.
• A detailed review of the pediatric relapsed/refractory neuroblastoma market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM pediatric relapsed/refractory neuroblastoma market.

Pediatric relapsed/refractory neuroblastoma Report Insights

• Patient Population
• Therapeutic Approaches
• Pediatric relapsed/refractory neuroblastoma Pipeline Analysis
• Pediatric relapsed/refractory neuroblastoma Market Size and Trends
• Existing and future Market Opportunity 

Pediatric relapsed/refractory neuroblastoma Report Key Strengths

• Ten-year Forecast
• 7MM Coverage 
• Pediatric Relapsed/Refractory Neuroblastoma Epidemiology Segmentation
• Key Cross Competition 
• Conjoint analysis
• Drugs Uptake and Key Market Forecast Assumptions

Pediatric Relapsed/Refractory Neuroblastoma Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs

• What is the historical and forecasted pediatric relapsed/refractory neuroblastoma patient pool/patient burden in the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan?
• Which combination of treatment approaches will have a significant impact on the pediatric relapsed/refractory neuroblastoma drug treatment market size?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
• What are the current and emerging options for the treatment of pediatric relapsed/refractory neuroblastoma? 
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies? 
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies? 
• How many key players are developing therapies for pediatric relapsed/refractory neuroblastoma?
• Which drug is the major contributor pediatric relapsed/refractory neuroblastoma market by 2034?

Reasons to buy

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the pediatric relapsed/refractory neuroblastoma market.
• Insights on patient burden/disease Incidence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• Highlights of access and reimbursement policies of approved therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.