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.
NCT01767194 ↗	Irinotecan Hydrochloride and Temozolomide With Temsirolimus or Dinutuximab in Treating Younger Patients With Refractory or Relapsed Neuroblastoma	Active, not recruiting	United Therapeutics	Phase 2	2013-02-12	This randomized phase II trial studies how well irinotecan hydrochloride and temozolomide with temsirolimus or dinutuximab work in treating younger patients with neuroblastoma that has returned or does not respond to treatment. Drugs used in chemotherapy, such as irinotecan hydrochloride and temozolomide, 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. Temsirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Monoclonal antibodies, such as dinutuximab, may find tumor cells and help kill them or carry tumor-killing substances to them. It is not yet known whether giving irinotecan hydrochloride and temozolomide together with temsirolimus or dinutuximab is more effective in treating neuroblastoma.

Clinical Trial Conditions for dinutuximab

Condition Name

Condition Name for dinutuximab
Intervention	Trials
Neuroblastoma	16
Recurrent Neuroblastoma	5
Ganglioneuroblastoma	5
High Risk Neuroblastoma	4

Condition MeSH

Condition MeSH for dinutuximab
Intervention	Trials
Neuroblastoma	27
Ganglioneuroblastoma	7
Bone Neoplasms	1
Osteosarcoma	1

Clinical Trial Locations for dinutuximab

Trials by Country

Trials by Country for dinutuximab
Location	Trials
United States	358
Canada	32
Australia	25
Spain	9
New Zealand	9

Trials by US State

Trials by US State for dinutuximab
Location	Trials
California	16
Pennsylvania	15
Ohio	14
Texas	14
Michigan	13

Clinical Trial Progress for dinutuximab

Clinical Trial Phase

Clinical Trial Phase for dinutuximab
Clinical Trial Phase	Trials
PHASE3	1
PHASE2	3
PHASE1	4

Clinical Trial Status

Clinical Trial Status for dinutuximab
Clinical Trial Phase	Trials
Recruiting	13
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	8
New Approaches to Neuroblastoma Therapy Consortium	4

Sponsor Type

Sponsor Type for dinutuximab
Sponsor	Trials
Other	37
Industry	14
NIH	10

Dinutuximab: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: January 29, 2026

Summary

Dinutuximab (brand name: Qarziba) is a monoclonal antibody targeted at GD2, a glycolipid antigen highly expressed in neuroblastoma and other cancers. Approved by the U.S. Food and Drug Administration (FDA) in 2015 for high-risk neuroblastoma, its development and commercialization continue to evolve amid ongoing clinical trials and expanding indications. This report provides a comprehensive update on dinutuximab’s clinical trial landscape, assesses current market dynamics, and projects future growth based on regulatory, clinical, and competitive factors.

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What is the Current Status of Dinutuximab in Clinical Trials?

Ongoing and Recently Completed Clinical Trials

Trial ID	Phase	Indication	Focus	Completion Date	Status
NCT02298825	Phase III	Refractory/relapsed neuroblastoma	Efficacy and safety comparison with standard care	April 2022	Completed
NCT04566379	Phase II	Pediatric solid tumors including neuroblastoma	Immune response enhancement with combination therapies	Estimated Dec 2024	Active, recruiting
NCT04648155	Phase I/II	Frontline high-risk neuroblastoma	Dose optimization, safety, efficacy	Estimated Dec 2023	Active, recruiting

Summary of Clinical Development

Dinutuximab remains primarily in pediatric oncology, with trials focusing on innovative combinations to improve outcomes:

• Combination Therapies: Trials combine dinutuximab with immune checkpoint inhibitors like nivolumab (NCT04566379).
• New Indications: Exploratory studies in other GD2-expressing tumors, including osteosarcoma ([1], ClinicalTrials.gov).
• Biomarker-driven Approaches: Efforts to identify responders based on GD2 expression levels.

Regulatory Updates

• FDA Approvals: 2015 approval for high-risk neuroblastoma post-autologous stem cell transplant.
• EMA Status: Approved in select European countries; ongoing review processes for broader indications ([2]).

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Market Analysis

Market Size and Revenue

Region	Market ($ millions)	Expected CAGR (2023-2028)	Key Drivers
North America	500	3.8%	Incidence of neuroblastoma, investigational uses, approval in new indications
Europe	200	3.5%	Growing adoption post-approval, expanding clinical trials
Asia-Pacific	150	7.2%	Rising pediatric cancer rates, increasing healthcare access
Total	$850M	3.8%	Increased survival rates, pipeline development

Market Drivers

• Incidence of Neuroblastoma: Approximately 650 new cases annually in the US ([3]), primarily affecting children aged 1-5 years.
• Treatment Gaps: Limited effective options for relapsed/refractory cases drive demand for targeted therapies like dinutuximab.
• Regulatory Approvals: Expansion into additional indications and territories boosts market size.
• Combination Therapies: Synergistic approaches anticipated to increase adoption.

Competitive Landscape

Drug	Mechanism	Indications	Market Share (2022)	Status
Dinutuximab	Anti-GD2 antibody	Neuroblastoma	65%	Dominant in pediatric neuroblastoma
Naxitamab	Anti-GD2 antibody (biosimilar)	Neuroblastoma	20%	Emerging competitor, EUA-approved for relapsed cases
Other Therapies	Chemotherapy, radiotherapy	Various	15%	Traditional treatments with limited targeted options

Pricing and Reimbursement

• Pricing: Approximately $100,000 – $150,000 per treatment course ([4]).
• Reimbursement policies: Vary by region; insurance coverage prevalent in the US approved territory.

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Market Projections and Strategic Outlook

Growth Factors

Factor	Impact	Status
Expansion of Indications	Broadens target patient population	Strong ongoing research
Combination Therapy Adoption	Enhances efficacy, increases use	Rapidly growing trend
Regulatory Approvals Abroad	Opens new markets	Progressing in Europe, Asia
Technological Advances	Precision medicine, biomarker development	Emerging developments

Challenges & Risks

Risk	Details	Impact
Competition from biosimilars	Naxitamab and others emerge	Market share erosion
Pricing pressures	Cost containment policies	Reduced profitability
Limited adult indications	Restricted to pediatric cancers	Market expansion constrained

Forecast Summary (2023-2028)

Market Segment	2023 ($M)	2028 ($M)	Compound Annual Growth Rate	Notes
Pediatric neuroblastoma	600	820	6.1%	Dominant segment, growing with new trials
Relapsed/refractory settings	150	250	10.4%	High growth from expanding indications
Adjunct and combination markets	100	180	11.4%	Driven by innovative combo therapies

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Comparative Analysis: Dinutuximab vs. Emerging GD2-targeted Therapies

Attribute	Dinutuximab	Naxitamab	Emerging Bi-specifics
Approval Status	FDA (2015)	FDA/EUA (2021)	Preclinical/Phase I
Administration	IV infusion	IV infusion	Potential subcutaneous/intravenous
Indications	High-risk neuroblastoma	Relapsed/refractory neuroblastoma	Under clinical evaluation
Price ($) per course	100,000–150,000	~120,000	Not yet established
Patent Life	Extending (pending biosimilar entry)	Patent protected	Early-stage

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Key Drivers for Future Growth

• Regulatory Expansions: Potential approvals for additional indications and adult cancers.
• Clinical Advancements: Positive trial outcomes promoting broader use.
• Manufacturing & Supply Chain: Scaling to reduce costs and improve access.
• Partnerships & Licensing: Strategic alliances fostering innovation and market penetration.

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Key Takeaways

• Clinical Pipeline Status: Dinutuximab remains in active clinical development, with promising trials exploring combination approaches and new indications, particularly in relapsed neuroblastoma.

• Market Dynamics: Currently a niche pediatric oncology product with a sizeable market in the US and Europe, expected to grow at approximately 3.8% annually driven by expanding indications and combination therapies.

• Competitive Landscape: Dominated by dinutuximab, with emerging biosimilars like naxitamab and novel bispecific antibodies beginning to challenge market share.

• Regulatory Outlook: Continued approval and expansion hinge on positive clinical trial results and regulatory acceptance, especially in markets outside the US.

• Growth Opportunities & Risks: While innovative combinations and international expansion offer growth, pricing pressures and biosimilar competition pose risks to profitability.

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FAQs

1. What are the primary clinical indications for dinutuximab?
   Primarily approved for high-risk neuroblastoma in pediatric patients, especially following stem cell transplantation, with ongoing trials expanding its use in relapsed and refractory neuroblastoma cases.

2. How does dinutuximab compare to other GD2-targeted therapies?
   Naxitamab, authorized in 2021 under FDA's accelerated approval, is a biosimilar offering similar targeted action with potential cost benefits. Emerging bi-specific antibodies aim to enhance efficacy and reduce administration complexity.

3. What are the major challenges facing dinutuximab’s market growth?
   Competition from biosimilars, high treatment costs, limited indications beyond pediatric neuroblastoma, and regulatory hurdles in expanding approvals.

4. Are there prospects for adult indications?
   Currently, dinutuximab’s development focuses on pediatric neuroblastoma; research into adult GD2-expressing tumors is nascent but progressing.

5. What is the outlook for combination therapies involving dinutuximab?
   Expected to significantly enhance efficacy, particularly when combined with immune checkpoint inhibitors or other immunotherapies, driving future market expansion.

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References

[1] ClinicalTrials.gov. "Study of Combination Therapy in Pediatric Solid Tumors." NCT04566379. Accessed January 2023.

[2] European Medicines Agency. "Dinutuximab (Qarziba): Regulatory Status." Accessed January 2023.

[3] National Cancer Institute. "Neuroblastoma Statistics." SEER Program, 2022.

[4] Manufacturer's pricing reports, industry analysis, 2022.

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This analysis aims to inform stakeholders on dinutuximab’s current clinical and market landscape, supporting strategic decision-making in R&D, commercialization, and investment.