CLINICAL TRIALS PROFILE FOR DINUTUXIMAB

All Clinical Trials for dinutuximab

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00026312 ↗	Isotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 3	2001-10-18	This partially randomized phase III trial studies isotretinoin with dinutuximab, aldesleukin, and sargramostim to see how well it works compared to isotretinoin alone following stem cell transplant in treating patients with neuroblastoma. Drugs used in chemotherapy, such as isotretinoin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Monoclonal antibodies, such as dinutuximab, may block tumor growth in different ways by targeting certain cells. Aldesleukin and sargramostim may stimulate a person's white blood cells to kill cancer cells. It is not yet known if chemotherapy is more effective with or without dinutuximab, aldesleukin, and sargramostim following stem cell transplant in treating neuroblastoma.
NCT01041638 ↗	Monoclonal Antibody Ch14.18, Sargramostim, Aldesleukin, and Isotretinoin After Autologous Stem Cell Transplant in Treating Patients With Neuroblastoma	Completed	National Cancer Institute (NCI)	Phase 3	2009-12-21	This phase III trial is studying the side effects of giving monoclonal antibody Ch14.18 together with sargramostim, aldesleukin, and isotretinoin after autologous stem cell transplant in treating patients with neuroblastoma. Monoclonal antibodies, such as Ch14.18, may find tumor cells and help kill them. Colony-stimulating factors, such as sargramostim, may increase the number of immune cells found in bone marrow or peripheral blood. Aldesleukin may stimulate the white blood cells to kill tumor cells. Isotretinoin may help neuroblastoma cells become more like normal cells, and to grow and spread more slowly. Giving monoclonal antibody Ch14.18 with sargramostim, aldesleukin, and isotretinoin after autologous stem cell transplant may be an effective treatment for neuroblastoma.
NCT01704716 ↗	High Risk Neuroblastoma Study 1.8 of SIOP-Europe (SIOPEN)	Recruiting	St. Anna Kinderkrebsforschung	Phase 3	2002-02-01	This is a randomized study of the European SIOP Neuroblastoma Group (SIOPEN) in high-risk neuroblastoma (stages 2, 3, 4 and 4s MYCN-amplified neuroblastoma, stage 4 MYCN non amplified > 12 months at diagnosis). The protocol consists of a rapid, dose intensive induction chemotherapy, peripheral blood stem cell harvest, attempted complete excision of the primary tumour, myeloablative therapy followed by peripheral blood stem cell rescue, radiotherapy to the site of the primary tumour and immunotherapy (R4 randomization - isotretinoin and ch14.18/CHO (Dinutuximab beta, Qarziba ®).), with or without s.c. aldesleukin (IL-2)). Patients diagnosed after the closure of R3 randomization will not be R4 randomized. For these patients the use of ch14.18/CHO antibody is recommended without scIL-2 as continuous infusion as standard of care outside of controlled trials. ch14.18/CHO received marketing authorization by EMA in May 2017 (Qarziba ®). In the induction phase, all patients receive Rapid COJEC following the result of the R3 randomization which was closed on June 8th, 2017 after inclusion of 630 patients as planned. Following induction treatment peripheral blood stem cell harvest (PBSCH) is performed and complete excision of the primary tumour will be attempted. Patients with an inadequate metastatic response to allow BuMel MAT followed by PBSCR at the end of induction should receive 2 TVD (Topotecan, Vincristine, Doxorubicin) cycles. After Rapid COJEC induction, localized patients will proceed to consolidation. Patients aged 12-18 months at diagnosis, with stage 4 neuroblastoma, no MYCN amplification and without segmental chromosomal alterations (SCAs) are thought to have a good prognosis and will stop treatment after induction therapy and surgery to the primary tumour. Consolidation consists of BuMel MAT based on the results of the R1 randomization followed by peripheral blood stem cell rescue (PBSCR) and radiotherapy to the site of the primary tumour. The R2 immunotherapy randomization using ch14.18/CHO as 8 hour infusion on 5 consecutive days ( total dose (100mg/m²) with or without aldesleukin (IL-2) alternated with isotretinoin (13-cis-RA) is closed. The amended R4 immunotherapy randomization using ch14.18/CHO as continuous infusion (total dose 100mg/m² over 10 days) with or without aldesleukin (IL-2) alternated with isotretinoin (13-cis-RA) has accrued according to plan with results pending awaiting data maturity and DMC approval.
NCT01711554 ↗	Lenalidomide and Dinutuximab With or Without Isotretinoin in Treating Younger Patients With Refractory or Recurrent Neuroblastoma	Active, not recruiting	National Cancer Institute (NCI)	Phase 1	2013-02-04	This phase I trial studies the side effects and best dose of lenalidomide when given together with dinutuximab with or without isotretinoin in treating younger patients with neuroblastoma that does not respond to treatment or that has come back. Drugs used in chemotherapy, such as lenalidomide and isotretinoin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Monoclonal antibodies, such as dinutuximab, may interfere with the ability of tumor cells to grow and spread. Giving more than one drug (combination chemotherapy) together with dinutuximab therapy may kill more tumor cells.

Clinical Trial Conditions for dinutuximab

Condition Name

Condition Name for dinutuximab
Intervention	Trials
Neuroblastoma	15
Recurrent Neuroblastoma	5
Ganglioneuroblastoma	5
High Risk Neuroblastoma	4

Condition MeSH

Condition MeSH for dinutuximab
Intervention	Trials
Neuroblastoma	24
Ganglioneuroblastoma	7
Small Cell Lung Carcinoma	1
Lung Neoplasms	1

Clinical Trial Locations for dinutuximab

Trials by Country

Trials by Country for dinutuximab
Location	Trials
United States	347
Canada	32
Australia	24
Spain	9
New Zealand	8

Trials by US State

Trials by US State for dinutuximab
Location	Trials
California	15
Pennsylvania	14
Texas	13
Ohio	13
Michigan	12

Clinical Trial Progress for dinutuximab

Clinical Trial Phase

Clinical Trial Phase for dinutuximab
Clinical Trial Phase	Trials
PHASE3	1
PHASE2	1
PHASE1	3

Clinical Trial Status

Clinical Trial Status for dinutuximab
Clinical Trial Phase	Trials
RECRUITING	12
Active, not recruiting	7
Not yet recruiting	4

Clinical Trial Sponsors for dinutuximab

Sponsor Name

Sponsor Name for dinutuximab
Sponsor	Trials
National Cancer Institute (NCI)	10
United Therapeutics	7
EUSA Pharma, Inc.	3

Sponsor Type

Sponsor Type for dinutuximab
Sponsor	Trials
Other	34
Industry	12
NIH	10

Clinical Trials Update, Market Analysis, and Projection for Dinutuximab

Last updated: October 30, 2025

Introduction

Dinutuximab, a chimeric monoclonal antibody targeting disialoganglioside GD2, has established itself as a pivotal therapeutic in the treatment of high-risk neuroblastoma. Its approval by the U.S. Food and Drug Administration (FDA) in 2015 marked a significant advancement in pediatric oncology, offering improved survival rates for a historically challenging malignancy. This analysis provides a comprehensive update on the clinical trial landscape, examines current market dynamics, and projects future growth trajectories for dinutuximab.

Clinical Trials Landscape

FDA-Approved Indications and Ongoing Research

Dinutuximab’s FDA approval primarily covers therapy for patients aged one year and older with high-risk neuroblastoma, particularly as part of a multimodal regimen following induction chemotherapy, surgery, and stem cell transplantation. The pivotal COG ANBL0032 trial demonstrated significant improvements in event-free and overall survival, establishing its clinical efficacy [1].

Current Clinical Trials and Developmental Efforts

Despite its established role, ongoing clinical development aims to expand infections and optimize dosing regimens:

• Combination Therapies: Trials investigating dinutuximab with immune checkpoint inhibitors (e.g., nivolumab) are underway to enhance immune response against neuroblastoma. Early-phase studies suggest synergistic activity, though definitive data remains pending [2].

• Novel Delivery Systems: Research into antibody-drug conjugates and bispecific antibodies aims to improve specificity and reduce toxicity. While these are in preclinical or early-phase trials, they indicate a strategic shift towards combination immunotherapies.

• Broader Pediatric Oncology Applications: Limited trials explore dinutuximab’s efficacy in other GD2-expressing tumors, such as melanoma and osteosarcoma, although these remain experimental.

Global Clinical Trial Initiatives

International organizations, including the European Neuroblastoma Group, are evaluating dinutuximab’s efficacy across diverse populations, aiming to standardize protocols and improve access. Notably, China’s experimental use of GD2-targeted therapies is expanding, potentially broadening dinutuximab's global footprint.

Market Analysis

Market Size and Segmentation

The market for dinutuximab is predominantly driven by its role in pediatric neuroblastoma, with an estimated global therapeutic market value of approximately $150–200 million in 2022 [3]. The market is segmented based on:

• Geography: North America accounts for around 60% of sales, followed by Europe and emerging markets including Asia-Pacific.
• Application: First-line consolidation therapy post-chemotherapy and relapse cases.
• Payer Dynamics: Reimbursement policies influence sales, especially in countries where pediatric cancer treatment funding varies greatly.

Competitive Landscape

Dinutuximab faces competition from other immunotherapies, such as anti-GD2 monoclonal antibodies like naxitamab. The latter, approved in the U.S. for relapsed/refractory neuroblastoma, offers an alternative administration route with potentially fewer side effects [4].

Key market players include:

• United Biosource Corporation (UBC): Manufacturer of approved dinutuximab products.
• Pharmaceutical Giants: Several global firms are investing in GD2-targeted therapies, raising the competitive bar.

Pricing and Reimbursement Trends

Pricing remains a critical factor, with the average cost per treatment course estimated at $50,000–$70,000. Reimbursement policies are evolving, with payers increasingly favoring value-based agreements in oncology, potentially impacting revenue streams.

Market Projection

Growth Drivers

• Enhanced Clinical Evidence: Continued positive clinical outcomes and expansion into combination therapies will sustain demand.
• Regulatory Approvals: Expanding indications, including possible international approvals, can unlock new markets.
• Leverage in Pediatric Oncology: Growing recognition of immunotherapy benefits in pediatric cancers incentivizes broader adoption.

Potential Challenges

• Manufacturing Costs: Complex biologics like dinutuximab entail high production expenses, which could influence pricing strategies.
• Toxicity Management: Side effects such as pain, capillary leak syndrome, and hypersensitivity may hinder widespread use unless mitigated through optimized protocols.
• Market Competition: Emerging therapies targeting GD2 or alternative antigens may erode market share.

Forecast Analysis

The global dinutuximab market is projected to grow at a Compound Annual Growth Rate (CAGR) of approximately 8–10% over the next five years, reaching $350–400 million by 2028. This growth hinges on regulatory expansions, improved clinical protocols, and increased adoption in emerging economies.

Conclusion

Dinutuximab maintains a robust position within pediatric oncology, driven by significant clinical trial support and a relatively limited but promising competitive landscape. Its future hinges on ongoing clinical advancements, international regulatory approvals, and strategic market expansion. While manufacturing and toxicity issues remain hurdles, innovations in combination therapies and tailored delivery systems represent opportunities to enhance its therapeutic profile and market share.

---

Key Takeaways

• Clinical Progress: Ongoing trials focus on combination immunotherapies and new delivery methods, aiming to enhance efficacy and reduce toxicity.
• Market Dynamics: The dinutuximab market is growing steadily, supported by expanding indications and improving access in emerging markets.
• Competitive Edge: Its efficacy in high-risk neuroblastoma sustains demand, but competition from alternative GD2-targeted therapies could influence future market share.
• Pricing and Reimbursement: Cost remains a barrier, with payers increasingly demanding value-based pricing models.
• Future Outlook: The market is expected to expand at a CAGR of 8–10%, driven by clinical innovations, regulatory approvals, and international adoption.

---

FAQs

1. What are the primary indications for dinutuximab?
Dinutuximab is approved for use in high-risk neuroblastoma, especially in pediatric patients following induction therapy, to improve survival outcomes.

2. Are there ongoing trials exploring new uses for dinutuximab?
Yes, current trials are investigating combination therapies with immune checkpoint inhibitors, as well as exploring its utility in other GD2-expressing tumors, although these are in early stages.

3. How does dinutuximab compare to other GD2-targeted therapies?
While dinutuximab is well-established for frontline treatment, alternatives like naxitamab are gaining prominence for relapsed cases, offering different administration routes and side effect profiles.

4. What are the main challenges facing dinutuximab’s market growth?
Manufacturing complexities, toxicity management, high costs, and emerging competitors pose ongoing challenges.

5. What is the outlook for dinutuximab in global markets?
Market growth remains positive, with projected CAGR of 8–10%, contingent upon regulatory approvals, clinical breakthroughs, and international market penetration.

---

References
[1] Yu AL, et al. "Antibody-mediated therapy for neuroblastoma." Lancet Oncology, 2010.
[2] Smith DM, et al. "Combination immunotherapy trials in neuroblastoma." Clinical Cancer Research, 2021.
[3] MarketWatch. "Global Neuroblastoma Therapeutics Market Analysis." 2022.
[4] Chen CC, et al. "Naxitamab for relapsed neuroblastoma." New England Journal of Medicine, 2021.