Pediatric Relapsed/Refractory Neuroblastoma Market is on an Upward Trend, Driven by Innovation and Unmet Need | DelveInsight

The outlook for the neuroblastoma treatment market is optimistic, fueled by progress in targeted therapies and immunotherapies. GD2-targeted immunotherapy is increasingly being incorporated into standard treatment regimens for patients who have not achieved remission following initial chemotherapy. Additionally, research is exploring the role of these immunotherapies in strengthening and maintaining the response achieved through chemotherapy.

LAS VEGAS, Aug. 14, 2025 /PRNewswire/ -- DelveInsight's Pediatric Relapsed/Refractory Neuroblastoma Market Insights report includes a comprehensive understanding of current treatment practices, pediatric relapsed/refractory neuroblastoma emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Takeaways from the Pediatric Relapsed/Refractory Neuroblastoma Market Report

• According to DelveInsight's analysis, the market size for pediatric relapsed/refractory neuroblastoma in the 7MM is expected to grow significantly by 2034.
• The United States accounted for the highest pediatric relapsed/refractory neuroblastoma treatment market size in 7MM in 2024, in comparison to the other major markets, i.e., EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• 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.
• Neuroblastoma occupies nearly 8% of pediatric malignancies and occurs in ~150–200 children each year in Japan.
• Leading pediatric relapsed/refractory neuroblastoma companies developing emerging therapies, such as Clarity Pharmaceuticals, Adaptimmune, Peel Therapeutics, Cellectar Biosciences, and others, are developing new pediatric relapsed/refractory neuroblastoma treatment drugs that can be available in the pediatric relapsed/refractory neuroblastoma market in the coming years.
• The promising pediatric relapsed/refractory neuroblastoma therapies in the pipeline include ⁶⁴Cu SARTATE/ ⁶⁷Cu SARTATE, Afamitresgene autoleucel, PEEL-224, Iopofosine I-131, and others.

Discover the pediatric relapsed/refractory neuroblastoma new treatment @ New Treatments for Pediatric Relapsed/Refractory Neuroblastoma

Pediatric Relapsed/Refractory Neuroblastoma Market Dynamics

The pediatric relapsed/refractory neuroblastoma market dynamics are expected to change in the coming years. The pediatric relapsed/refractory neuroblastoma market is primarily driven by the high unmet medical need and limited treatment options available for this aggressive and recurrent form of cancer. Advances in precision medicine, immunotherapy, and targeted drug development are catalyzing investment and innovation in this space. Increased awareness and improved diagnostic capabilities are enabling earlier detection and more accurate disease stratification, which supports the adoption of novel therapies. Moreover, regulatory incentives such as orphan drug designation and pediatric priority review vouchers are encouraging biopharmaceutical companies to invest in developing therapies for this rare pediatric population.

As potential therapies are being investigated for the treatment of pediatric relapsed/refractory neuroblastoma, it is safe to predict that the treatment space will significantly impact the pediatric relapsed/refractory neuroblastoma market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the pediatric relapsed/refractory neuroblastoma market in the 7MM.

However, several factors may impede the growth of the pediatric relapsed/refractory neuroblastoma market. One major challenge is the limited patient population, as neuroblastoma is a rare pediatric cancer, and relapsed/refractory cases represent an even smaller subset, making it difficult to conduct large-scale clinical trials and attract substantial investment. Additionally, the biological complexity and heterogeneity of the disease contribute to poor response rates and rapid resistance to current treatments. Regulatory hurdles, including stringent pediatric trial requirements and ethical considerations, further complicate drug development. Moreover, there is a lack of validated biomarkers and standardized endpoints, which impedes the design and evaluation of novel therapies.

Moreover, pediatric relapsed/refractory neuroblastoma treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the pediatric relapsed/refractory neuroblastoma market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the pediatric relapsed/refractory neuroblastoma market growth.

Pediatric Relapsed/Refractory Neuroblastoma Treatment Market

Current treatment options for pediatric patients with relapsed or refractory neuroblastoma include surgery, chemotherapy, radiation therapy, and stem cell transplantation. Several drugs have been approved for this indication, including IWILFIN (developed by USWM and Norgine), DANYELZA (Y-mAbs Therapeutics), and QARZIBA (EUSA Pharma), among others.

IWILFIN is an oral prescription therapy used to reduce the risk of relapse in both adults and children with high-risk neuroblastoma who have shown a response to previous multi-modal treatments. Its active ingredient, eflornithine, irreversibly inhibits the enzyme Ornithine Decarboxylase (ODC)—a key enzyme in polyamine biosynthesis and a transcriptional target of the oncogene MYCN. Polyamines are essential for cellular proliferation and survival, particularly in cancer cells. By blocking polyamine synthesis, eflornithine helps rebalance the LIN28/Let-7 pathway, which regulates cancer stem cells and glycolytic metabolism. This leads to downregulation of MYCN and LIN28B, particularly in MYCN-amplified neuroblastoma, thereby inhibiting tumor growth.

In July 2021, US WorldMeds entered into an exclusive licensing agreement with Norgine to commercialize eflornithine in Europe. Subsequently, in December 2023, the FDA approved IWILFIN for use in adult and pediatric patients with high-risk neuroblastoma who had at least a partial response to previous combination therapies, including GD2-targeted immunotherapy. This approval was based on data from an externally controlled trial comparing Study IIIb (treatment arm) with Study ANBL0032 (control). A marketing authorization application for IWILFIN was submitted to the European Medicines Agency (EMA) in January 2025.

DANYELZA is a GD2-targeting monoclonal antibody indicated for pediatric patients aged one year and older, as well as adults, with relapsed or refractory high-risk neuroblastoma involving the bone or bone marrow. It is used in combination with GM-CSF for patients who have shown at least a stable disease, minor response, or partial response to prior treatments. GD2, a disialoganglioside, is highly expressed on neuroblastoma cells and other neuroectoderm-derived tissues. The antibody naxitamab-gqgk, the active component in DANYELZA, mediates complement-dependent cytotoxicity (CDC) and antibody-dependent cell-mediated cytotoxicity (ADCC) by binding to GD2.

The FDA approved DANYELZA in November 2020 for patients aged one and older with relapsed/refractory neuroblastoma in the bone or bone marrow, based on findings from Study 201 and Study 12-230.

In November 2024, Y-mAbs Therapeutics announced an exclusive licensing and distribution agreement with Nobelpharma for the development and commercialization of DANYELZA in Japan for high-risk neuroblastoma, with a potential future extension to relapsed osteosarcoma. Under this agreement, Nobelpharma will lead regulatory submissions and oversee marketing, sales, and distribution in Japan upon regulatory approval.

To know more about pediatric relapsed/refractory neuroblastoma treatment options, visit @ Approved Pediatric Relapsed/Refractory Neuroblastoma Drugs

Pediatric Relapsed/Refractory Neuroblastoma Pipeline Therapies and Key Companies

The pipeline consists of products such as ⁶⁴Cu SARTATE/ ⁶⁷Cu SARTATE (Clarity Pharmaceuticals), TECELRA (afamitresgene autoleucel) (Adaptimmune), PEEL-224 (Peel Therapeutics), and others.

SARTATE is an advanced, next-generation theranostic radiopharmaceutical designed with enhanced specificity and improved in-vivo stability. It targets cancers expressing Somatostatin Receptor 2 (SSTR2) and is being developed for use in diagnosis, staging, and subsequent treatment of these cancers.

Clarity Pharmaceuticals is currently conducting a Phase I/II clinical trial to assess the safety and efficacy of ⁶⁷Cu-SARTATE in pediatric patients with high-risk neuroblastoma. The trial has progressed to Cohort III. Both ⁶⁷Cu-SARTATE and ⁶⁴Cu-SARTATE have received Orphan Drug Designation (ODD) from the U.S. FDA for the treatment and clinical management of neuroblastoma. Additionally, the FDA has granted Rare Pediatric Disease Designation (RPDD) to ⁶⁷Cu-SARTATE for the treatment of neuroblastoma.

PEEL-224 is a nanocarrier-based therapy linked to four small molecules, developed as a cancer treatment. Its active agent is derived from camptothecin, a natural compound from the Chinese Happy Tree, which is believed to have evolved as a plant defense mechanism. Preclinical results have been highly encouraging, demonstrating that PEEL-224 can achieve sustained complete tumor responses in aggressive solid tumors, in some instances lasting over six months.

PEEL-224 is currently being evaluated in a Phase I/II clinical trial for solid tumors, including a neuroblastoma cohort comprising patients aged 1 to 30 years.

The anticipated launch of these emerging therapies are poised to transform the pediatric relapsed/refractory neuroblastoma market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the pediatric relapsed/refractory neuroblastoma market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.

Discover more about pediatric relapsed/refractory neuroblastoma marketed and pipeline drugs @ Pediatric Relapsed/Refractory Neuroblastoma Clinical Trials

Recent Developments in the Pediatric Relapsed/Refractory Neuroblastoma Market

• 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 Overview

Neuroblastoma is a type of cancer that arises from immature nerve cells, known as neuroblasts, typically in infants and young children, most often before the age of five. In many cases, it begins during infancy and can even be detected within the first month of life. While it commonly originates in the adrenal glands, it can also form in other areas such as the neck, chest, or along the spine. This cancer is considered aggressive because it frequently spreads to other parts of the body by the time it is diagnosed.

Symptoms can vary but may include a noticeable lump at the tumor site, bone pain, gastrointestinal issues like diarrhea, and neurological problems. The underlying cause of neuroblastoma is usually unknown, although in rare cases, it may result from inherited genetic mutations, such as those in the ALK or PHOX2B genes.

Advances in research have significantly improved survival rates, but challenges remain. About 10% to 15% of children with high-risk neuroblastoma do not respond to initial therapy, and approximately 40% to 50% experience a return of the disease after treatment.

Diagnosis involves a thorough evaluation, including physical examination, blood tests, imaging techniques such as MRI and CT scans, and confirmation via biopsy. Two main staging systems are used to classify the disease: the International Neuroblastoma Risk Group Staging System (INRGSS), which is based on imaging before treatment (L1, L2, M, MS), and the International Neuroblastoma Staging System (INSS), which uses surgical findings (Stages I–IV, 4S). Risk assessment under the INRG framework incorporates both clinical and genetic factors, such as MYCN gene amplification, chromosome 11q status, and tumor cell ploidy, to categorize tumors into very low-, low-, intermediate-, or high-risk groups.

Pediatric Relapsed/Refractory Neuroblastoma Epidemiology Segmentation

The pediatric relapsed/refractory neuroblastoma epidemiology section provides insights into the historical and current pediatric relapsed/refractory neuroblastoma patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.

The pediatric relapsed/refractory neuroblastoma market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into:

• Total Incident Cases of Neuroblastoma
• Total Incident Cases of Pediatric Relapsed/Refractory Neuroblastoma
• Incident Cases of Pediatric Relapsed/Refractory Neuroblastoma By Risk Groups
• Total Treatable Cases of Pediatric Relapsed/Refractory Neuroblastoma

Scope of the Pediatric Relapsed/Refractory Neuroblastoma Market Report

• Therapeutic Assessment: Pediatric Relapsed/Refractory Neuroblastoma current marketed and emerging therapies
• Pediatric Relapsed/Refractory Neuroblastoma Market Dynamics: Key Market Forecast Assumptions of Emerging Pediatric Relapsed/Refractory Neuroblastoma Drugs and Market Outlook
• Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Unmet Needs, KOL's views, Analyst's views, Pediatric Relapsed/Refractory Neuroblastoma Market Access and Reimbursement

Download the report to understand which factors are driving pediatric relapsed/refractory neuroblastoma therapeutics market trends @ Pediatric Relapsed/Refractory Neuroblastoma Market Trends

Table of Contents

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