Neuroblastoma Market
- The Neuroblastoma Treatment Market is anticipated to increase with a notable CAGR during the forecast period, 2024–2034
- About 90% of Neuroblastoma is found in children younger than 5. 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 available Neuroblastoma Treatments include surgery, chemotherapy, radiation therapy, and stem cell transplantation.
- Several approved Neuroblastoma Drugs include IWILFIN (USWM), DANYELZA (Y-mAbs Therapeutics), QARZIBA (EUSA Pharma), and others.
- Eflornithine (IWILFIN) received FDA approval to reduce the risk of relapse in pediatric and adult patients with high-risk neuroblastoma, making it the first therapy to minimize the risk for relapse in children with the disease.
- Norgine B.V. announced its first marketing authorization application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma, via Project Orbis in Australia, Switzerland, and the United Kingdom.
- The Neuroblastoma Pipeline is very potent, with many emerging drugs such as 64Cu SARTATE/ 67Cu SARTATE, idasanutlin, SACT-1, LEE011, and others.
- In April 2024, Essential Pharma completed the acquisition of the entire issued share capital of Renaissance Pharma with its lead asset being Hu14.18K322A (Hu14.18), an immunotherapy currently in Phase II clinical development for the treatment of high-risk neuroblastoma (HRNB).
- In June 2024, Essential Pharma signed a strategic agreement with AGC Biologics. AGC Biologics will produce Hu1418K322A (Hu14.18).
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DelveInsight's “Neuroblastoma Cancer Market Insight, Epidemiology and Market Forecast – 2034” report delivers an in-depth analysis of neuroblastoma epidemiology, market, and clinical development in neuroblastoma. In addition to this, the report provides historical and forecasted epidemiology and market data as well as a detailed analysis of the neuroblastoma market trends in the United States, EU4 (Germany, France, Italy, and Spain ), the United Kingdom, and Japan.
Neuroblastoma Cancer 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 neuroblastoma market size from 2020 to 2034 in 7MM. The report also covers current Neuroblastoma Treatment Market practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s underlying potential.
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Study Period |
2020–2034 |
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Forecast Period |
2024–2034 |
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Geographies Covered |
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Neuroblastoma Epidemiology
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Segmented by
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Neuroblastoma Companies |
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Neuroblastoma Therapies |
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Neuroblastoma Market |
Segmented By
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Neuroblastoma Analysis |
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Neuroblastoma Cancer Treatment Market
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.
Neuroblastoma Diagnosis
The Neuroblastoma 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
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.
Neuroblastoma Epidemiology
The neuroblastoma epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented as total incident cases of neuroblastoma, age-specific incident cases of neuroblastoma, incident cases of neuroblastoma by risk groups, and treatable cases of neuroblastoma in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), and the United Kingdom, and Japan from 2020 to 2034.
- Among the 7MM, the US had the highest number of Neuroblastoma Incidence Cases in 2023.
- There are about 700–800 new cases of neuroblastoma each year in the US.
- Around 100 children between the ages of 0 and 14 years are diagnosed with neuroblastoma each year in the UK.
- Neuroblastoma occupies ~8% of pediatric malignancies and occurs in 150–200 children each year in Japan
Neuroblastoma Drugs Market Chapters
The drug chapter segment of the Neuroblastoma Therapeutics Market Report encloses a detailed analysis of neuroblastoma-marketed drugs and late-stage (Phase III and Phase II) Neuroblastoma Pipeline Drugs Analysis. It also deep dives into Neuroblastoma Clinical Trials details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations.
Neuroblastoma Marketed Drugs
- IWILFIN (eflornithine): USWM (US WorldMeds)
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 December 2023, the FDA approved IWILFIN to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy. The approval was based on efficacy, evaluated in an externally controlled trial comparing outcomes from Study IIIb (investigational arm) and Study ANBL0032.
- DANYELZA (naxitamab-gqgk): 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.
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Table 1: Comparison of Key Marketed Drugs | |||
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Drug Name |
Company |
MoA |
US Approval |
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IWILFIN |
USWM |
ODC inhibitor |
2023 |
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DANYELZA |
Y-mAbs Therapeutics |
GD2 receptor |
2020 |
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UNITUXIN |
United Therapeutics Corporation |
GD2 inhibitor |
2015 |
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QARZIBA |
EUSA Pharma |
GD2 inhibitor |
2017 |
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 Neuroblastoma Clinical Trials to evaluate the safety and efficacy of 67Cu-SARTATE in pediatric patients with high-risk neuroblastoma. The trial has been advanced to the Cohort III. 67Cu SARTATE and 64Cu SARTATE have been granted FDA Orphan Drug Designation status for the treatment and clinical management of neuroblastoma. The US FDA has also granted Rare Pediatric Disease Designation to 67Cu-SARTATE to treat neuroblastomas.
- SACT-1: Aptorum Group
SACT-1 is an orally administered repurposed small molecule drug to target neuroblastoma. SACT-1’s mechanism has been investigated in preclinical studies to enhance tumor cell death and suppress MYCN expression (a common clinical diagnosis in high-risk or relapsed neuroblastoma patients where an amplification of MYCN is usually observed). SACT-1 is designed to be used especially in combination with standard-of-care chemotherapy.
In June 2023, Aptorum submitted the relevant Phase Ib/IIa Neuroblastoma Clinical Trials protocol of SACT-1 to the US FDA. The Phase Ib/IIa study of SACT-1 submitted is for the combination with chemotherapy for first relapse or refractory high risk neuroblastoma. The US FDA granted Orphan Drug Designation to SACT-1 for the treatment of patients with Neuroblastoma.
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Table 2: Comparison of key emerging drugs | ||||
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Drug name |
Company |
MoA |
Phase |
Indication |
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SARTATE |
Clarity Pharmaceuticals |
SSTR2 inhibitor |
I/II |
Pediatric patients with high-risk neuroblastoma |
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LEE011 |
Novartis |
CDK4 and CDK6 inhibitor |
I/II |
Pediatric patients with relapsed or refractory neuroblastoma and other solid tumors |
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LY2835219 |
Eli Lilly |
CD4/6 inhibitor |
Ib/II |
Pediatric patients with relapsed or refractory neuroblastoma and other solid tumors |
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Idasanutlin |
Hoffmann-La Roche |
MDM2 antagonist |
I/II |
Pediatric and young adult patients with relapsed/refractory neuroblastoma |
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ch14.18 |
United Therapeutics |
Anti-GD2 |
I/II |
High-risk neuroblastoma |
Neuroblastoma Drugs Market Insights
- GD2 inhibitors
Neuroblastoma Market Outlook
- In 2023, the US accounted for the largest Pediatric Neuroblastoma Treatment Market, which is expected to grow during the forecast period.
- In December 2023, the FDA approved the first oral maintenance treatment for high-risk neuroblastoma i.e. IWILFIN.
- In August 2023, NorthStar Medical Radioisotopes announced that the first patient had been dosed in a Neuroblastoma Clinical Trials using NorthStar’s electron accelerator-produced Cu-67. Administration of the therapeutic dose took place as part of an ongoing Phase I/IIa theranostic clinical trial, conducted by Clarity Pharmaceuticals to investigate the safety and efficacy of Cu-67 SARTATE in pediatric patients with high-risk neuroblastoma.
- The study of LY2835219 (abemaciclib) in combination with other anti-cancer treatments in children and young adult participants is anticipated to complete Phase I/II by early 2025. Other emerging drugs like SARTATE and LEE011 are expected to complete their ongoing trials by 2028.
Neuroblastoma Drugs Uptake
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KOL Views |
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“Many new targeted therapies are under investigation, but the main finding of importance to childhood cancer may be the anti-GD2 antibody for neuroblastoma; and a little longer ago, rituximab for lymphoma and imatinib for Ph1+ leukemias. There have been many discoveries of genes involved in cancer initiation and progression, but it is too early to say what the impact on cure will be.” Head of the MIBG Program at UCSF |
Pediatric Neuroblastoma Market: Qualitative Analysis
We perform Qualitative and Pediatric Neuroblastoma 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.
Neuroblastoma Drugs 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 Neuroblastoma Drugs Market 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.
Pediatric Neuroblastoma Treatment Market Report Scope
- The Pediatric Neuroblastoma Treatment Market Report covers a segment of key events, an executive summary, a descriptive overview, 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 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 Pediatric Neuroblastoma Treatment Market landscape.
- A detailed review of the Pediatric Neuroblastoma Treatment Market, historical and forecasted Neuroblastoma Treatment Market Size, Neuroblastoma Drugs Market Share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
- The Pediatric Neuroblastoma Treatment Market 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 neuroblastoma market.
Neuroblastoma Cancer Market Report Insights
- Patient-based Neuroblastoma Market Forecasting
- Neuroblastoma Therapeutic Approaches
- Neuroblastoma Pipeline Drugs Analysis
- Neuroblastoma Cancer Market Size and Trends
- Existing and future Neuroblastoma Cancer Market Opportunity
Neuroblastoma Cancer Market Report Key Strengths
- 11 Years Neuroblastoma Cancer Market Forecast
- 7MM Coverage
- Neuroblastoma Epidemiology Segmentation
- Key Cross Competition
- Conjoint analysis
- Neuroblastoma Drugs Uptake
- Key Neuroblastoma Market Forecast Assumptions
Pediatric Neuroblastoma Treatment Market Report Assessment
- Current Pediatric Neuroblastoma Treatment Market Practices
- Neuroblastoma Unmet Needs
- Neuroblastoma Pipeline Drugs Profiles
- Neuroblastoma Cancer Market Attractiveness
- Qualitative Analysis (SWOT and Conjoint Analysis)
FAQs
- What is the historical and forecasted neuroblastoma patient pool/patient burden in the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan?
- Which combination Neuroblastoma Treatment approaches will have a significant impact on the Pediatric Neuroblastoma Treatment 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 neuroblastoma?
- What are the recent novel therapies, targets, Neuroblastoma 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 Neuroblastoma Companies are developing therapies for neuroblastoma?
- Which drug will be the major contributor to the neuroblastoma cancer market by 2034?
Reasons to Buy
- The Pediatric Neuroblastoma Treatment Market Report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the neuroblastoma cancer market.
- Insights on patient burden/disease: Neuroblastoma 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 Neuroblastoma Cancer 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 Neuroblastoma Cancer Market so that the upcoming players can strengthen their development and launch strategy.
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