The neuroblastoma therapeutic market is estimated to be valued at USD 2.78 Bn in 2024 and is expected to reach USD 4.44 Bn by 2031, growing at a compound annual growth rate (CAGR) of 6.9% from 2024 to 2031. Rising drug approvals and emerging pipeline drugs to treat neuroblastoma are further supporting the market growth. Manufacturers are continuously focusing on developing novel therapeutics with improved efficacy and safety against neuroblastoma.

Market Driver - Increasing Incidence of Neuroblastoma Among Children

One of the key drivers contributing to market growth that is evident is the rising incidence of this pediatric cancer among children globally. Researchers from hospitals and cancer centers in North America and Europe have reported a slow but steady rise in the number of neuroblastoma cases being diagnosed each year, with more infants and children now being affected by this cancer than ever before.

Furthermore, as diagnostic and screening capabilities are improving all over the world, especially in developing countries, more neuroblastomas are also being detected at an early stage which otherwise may have gone unnoticed before.

With a growing pediatric patient population susceptible to neuroblastoma, the demand for effective treatment options is amplified. Pharmaceutical companies recognize this public health issue and see a widening treatment gap. Consequently, they are allocating additional resources towards research and trials of newer medicines that can target the specific molecular abnormalities and pathways involved in neuroblastoma development, hopefully improving survival outcomes.

Several candidate drugs are currently in the pipeline at different stages of evaluation. If successful, these novel targeted agents and immunotherapies will generate a substantial new revenue stream in the coming years to fulfil this rising need.

Market Driver - Growing Investments in Research and Development of Targeted Therapies

Another important factor positively impacting the neuroblastoma therapeutic sector is the mounting investments from both public and private sources into R&D of more customized treatment approaches.

Major pharmaceutical corporations as well as small biotech startups are devoting greater resources into research of targeted drugs and cell/gene-based therapies. Academic medical centers worldwide are also fundraising more to support innovative investigator-initiated trials of novel agents and combinations. Compared to a decade ago, there is significantly higher activity today around identification of genetic aberrations that fuel neuroblastoma growth and testing of therapies directly counteracting those vulnerabilities. Grantmaking organizations are prioritizing neuroblastoma for awards to test precision oncology concepts in this disease.

With personalized medicine becoming mainstream in oncology, stakeholders clearly foresee major market potential for any targeted therapy that can demonstrate reliable, clinically-meaningful benefit against neuroblastoma, especially for high-risk or relapsed patients facing limited options. As a result, targeted therapeutic discovery and translation are witnessing an unprecedented surge of capital from profit-driven as well as nonprofit sources aiming to capitalize on this unmet need. This expanding R&D base will catapult the field ahead, ultimately providing physicians an armamentarium of novel precision agents that improves clinical management of neuroblastoma going forward.

Market Challenge - High Treatment Costs

The neuroblastoma therapeutic market faces significant challenges due to high treatment costs associated with developing new therapies for this rare childhood cancer. Neuroblastoma treatment often involves intensive chemotherapy, surgery, radiation therapy and stem cell transplants which makes it one of the costliest pediatric cancers to treat.

Developing new drugs also have astronomical R&D costs running into billions of dollars. This puts significant financial pressure on pharmaceutical companies and deters many from actively pursuing the development of novel neuroblastoma medicines.

The low demand for drugs in the small patient pool means drug makers are unable to achieve significant sales volumes needed to recover high development expenses. These costs ultimately get passed on to parents and families in the form of out-of-pocket copays and deductibles, making treatment unaffordable for many. In the absence of government support or subsidies, high drug prices pose a major barrier to access of life-saving neuroblastoma therapies.

Market Opportunity - Development of Precision Medicine and Immunotherapies

A major opportunity in the neuroblastoma therapeutic market is the ongoing development of precision medicine and immunotherapies. With advances in genomics and proteomics, researchers are gaining deeper understanding of the molecular subtypes and drivers of this cancer. This is enabling the design of targeted medicines that block specific genetic mutations fueling tumor growth.

Concurrently, cancer immunotherapies that harness the body's immune system to fight neuroblastoma cells are also being explored. Personalized vaccinations, immune checkpoint inhibitors, T-cell therapies and other cell-based approaches offer hope of developing more effective and longer lasting treatments.

If successful, these precision and immune-based therapies could significantly improve survival outcomes for subsets of high-risk patients. Their novel mechanisms of action may also help overcome resistance to chemotherapy. The neuroblastoma therapeutic market is anticipated to witness increased investment and uptake of these newer targeted and biological therapies in the coming years.

Neuroblastoma is a type of childhood cancer that develops from immature nerve cells. Treatment is dependent on the stage and risk level of the cancer. For low-risk disease (Stage 1/2/4S), close observation or corticosteroid therapy may be sufficient.

For intermediate or high-risk localized disease (Stage 2A/2B/3), chemotherapy is prescribed as the primary treatment. Common regimens include cisplatin/carboplatin plus etoposide or cyclophosphamide. Some prescribers prefer carboplatin over cisplatin due to its more favorable side effect profile. If there is a complete or very good partial response after chemotherapy, surgeons will then perform tumor resection to remove any remaining cancer sites.

For high-risk metastatic or recurrent disease (Stage 4), an intensive multi-agent chemotherapy and stem cell transplant is standard. Prescribers commonly use combinations such as temozolomide/irinotecan/cisplatin or topotecan/cyclophosphamide. Bone marrow-ablative chemotherapy with autologous stem cell rescue is also routinely prescribed to consolidate the response.

Prescribers consider factors like tumor burden, genetics/biology, organ dysfunction and prior treatment history when selecting chemotherapy regimens and transplant timing. Response to initial therapy also influences subsequent treatment decisions. Close monitoring is important as late recurrences within 5 years are not uncommon.

Neuroblastoma stages range from stage 1 to stage 4. Stage 4 is the most advanced stage where cancer has spread widely.

For stage 1 and 2, the main treatment is surgery to remove the tumor. For stage 3, multi-agent chemotherapy is given before and after surgery to reduce the risk of relapse. Common regimens include carboplatin, etoposide and cyclophosphamide in combination with drugs like doxorubicin. This intensifies the treatment effect.

For stage 4 and relapsed/refractory disease, high-dose chemotherapy followed by stem cell transplant is the standard option. Drugs like busulfan, melphalan and thiotepa are used as a part of the chemotherapy regimen to provide an intensive dose. This is then followed by autologous stem cell transplantation to rescue the bone marrow.

For refractory or relapsed patients post transplantation, antibody-drug conjugates like unituxin (dinutuximab), which links an anti-GD2 antibody to a chemotherapy drug have shown positive results. Combining it with interleukins like interleukin-2 boosts the immune system further to fight tumors.

Salvage chemotherapy using irinotecan, temozolomide or topotecan along with retinoic acid and immunotherapy contribute to better outcomes. Choice depends on prior treatments, tumor markers, and organ involvement. Intensive multimodality treatment with careful selection offers long-term survival benefits in high-risk neuroblastoma.

Clinical trial diversification and innovation: Companies like Pfizer and United Therapeutics have had success by pursuing diverse clinical trial strategies for their neuroblastoma drug candidates. Pfizer's dinutuximab saw accelerated approval in 2015 after positive results in a Phase 2 trial evaluating its use with IL-2 and GM-CSF for relapsed/refractory patients.

Biomarker-driven drug development: Targeting pathways like ALK has been an effective strategy for companies like Pfizer, Roche, and Novartis. Roche gained approval in 2018 for Alecensa (alectinib) for ALK-positive neuroblastoma after a trial showed superiority over chemotherapy. Pfizer is also studying alectinib. By focusing therapies on biomarkers that can predict response, like ALK, developers have drugs that work for carefully selected, well-defined subsets - making approvals easier to achieve.

Partnerships for drug combinations: Companies often collaborate to combine their drugs, as combination therapies may be required for neuroblastoma. For example, United Therapeutics partnered with MacroGenics in 2016 to evaluate treprostinil plus MGA012 (an anti-PD-1 mAb) based on their complementary mechanisms. Such partnerships allow companies to evaluate more combination strategies than anyone could do alone, increasing the chances of success.

Insights, By Treatment: Rising Prevalence of Cancer Drives the Chemotherapy Segment Growth

In terms of treatment, chemotherapy is expected to hold 60.1% share of the market in 2024, owning to the rising prevalence of cancer globally. Chemotherapy involves use of cytotoxic anti-neoplastic drugs to destroy cancerous cells and curb tumor growth. It remains one of the principal modalities for neuroblastoma treatment, prescribed either alone or in combination with surgery and radiation therapy depending on the stage and type of cancer.

Wide acceptance of chemotherapy especially for high-risk and relapsed cases works in its favor. Moreover, continual development of new chemotherapy regimens incorporating novel targeted agents and immunotherapies help enhance treatment efficacy, thereby positioning chemotherapy as a mainstay for neuroblastoma treatment in the years to come.

Insights, By Drug Development Stage: Focus on Orphan Drugs Boosts Phase II Clinical Trials Segment

Phase II clinical trials is projected to account for 45.3% share among the drug development stages segments in 2024, due to increasing focus of pharmaceutical companies on developing orphan drugs for rare cancers like neuroblastoma. Phase II trials aim to evaluate drug efficacy and short-term side effects in a patient group.

As neuroblastoma impacts a small patient population, sponsors get regulatory incentives and assistance to develop orphan drugs through clinical trials. Successful phase II trials further the candidate drug to phase III for expanded assessment. Industry participants are steadily advancing promising treatment candidates through phase II trials in combination with immunotherapies, targeted therapies and other modalities to fast-track new drugs to market.

Insights, By Molecule Type: Advantages over Small Molecules Drive Recombinant Fusion Proteins Segment

Recombinant fusion proteins command the largest share in the molecule type segment owing to their distinct advantages over small molecule drugs. Recombinant fusion proteins offer higher specificity, less adverse effects and ability to combine multiple mechanisms of action into one molecule to achieve improved clinical outcomes for neuroblastoma.

Additionally, recombinant fusion proteins can be designed to have prolonged half-lives in plasma through fusion to IgG regions, allowing lower and less frequent dosing compared to conventional therapies. These unique characteristics have positioned various recombinant fusion protein candidates like dinutuximab and lumretuzumab at the forefront of neuroblastoma treatment landscape.

Their ability to synergize with other treatment modalities while presenting a favorable safety profile is anticipated to propel long-term demand for recombinant fusion proteins over other drug classes.

• The report indicates that neuroblastoma is most commonly diagnosed in children under the age of five, accounting for 15% of all pediatric cancer deaths. It highlights the stark differences in survival outcomes between low-risk and high-risk patients.
• Neuroblastoma is responsible for over 600 annual cases in the United States, with higher prevalence in non-African-American populations. It often metastasizes to lymph nodes, bones, bone marrow, and liver.
• Advances in antibody-drug conjugates (ADCs) have paved the way for more effective neuroblastoma treatments, with Y-mAbs Therapeutics leading the charge in developing therapies that offer better precision and reduced toxicity compared to traditional chemotherapy.

The major players operating in the neuroblastoma therapeutic market include United Therapeutics, AUM Biosciences, Cellectar Biosciences, Y-mAbs Therapeutics, Seagen Inc., Clarity Pharmaceuticals, Recombio, Eli Lilly and Company, Aptorum Group, PersonGen BioTherapeutics, and Ascentage Pharma.

• Clarity Pharmaceuticals is conducting a Phase I/IIa clinical trial (CL04) using 67Cu-SARTATE, a theranostic drug targeting somatostatin receptor 2 (SSTR2)-expressing tumors, including high-risk neuroblastoma. This drug, which has received FDA Orphan Drug Designation, aims to improve diagnostic accuracy and therapeutic outcomes while potentially reducing side effects by precisely targeting cancer cells expressing SSTR2. The trial is being conducted at multiple sites in the U.S. to assess safety and efficacy​.
• In March 2024, Ascentage Pharma initiated a Phase I trial for its MDM2 inhibitor APG-115, targeting the p53-mediated apoptosis pathway. The drug has received multiple FDA Orphan Drug Designations, showing potential to treat neuroblastoma alongside other cancers. APG-115 has been in Phase I and II trials for various cancers, including melanoma and combinations with immunotherapies like pembrolizumab.
• In November 2022, AUM Biosciences received Orphan Drug Designation for AUM302 for the treatment of neuroblastoma. AUM302 is a first-in-class oral kinase inhibitor that targets kinases like PI3K, PIM, and mTOR, which are involved in neuroblastoma progression, and it is expected to improve treatment outcomes for patients with neuroblastoma.

• By Treatment
  • Chemotherapy
  • Immunotherapy
• By Drug Development Stage
  • Phase II
  • Phase I
  • Preclinical
  • Discovery
• By Molecule Type
  • Recombinant fusion proteins
  • Small molecules
  • Monoclonal antibodies
  • Peptides
  • Polymers
  • Gene therapy
• By Patient Population
  • Pediatric
  • Adolescents