Qarziba (dinutuximab beta) / BeOne Medicines, Jazz, Apeiron Biologics, Recordati - LARVOL DELTA

Single-Center Cohort of Pediatric Patients with High-Risk Neuroblastoma Receiving Immunotherapy. (PubMed, Cancers (Basel)) - "Our experiences confirm that immunotherapy, including Dinutuximab beta and Nivolumab, is safe and well tolerated. The standardization of the application of Dinutuximab beta and in combination with novel therapeutic agents in maintenance and refractory/relapsed cases may contribute to improved treatment outcome results."

Journal • Hypotension • Neuroblastoma • Oncology • Pediatrics • Solid Tumor

Irinotecan, temozolomide and naxitamab plus GM-CSF (HITS) and naxitamab plus GM-CSF and ifosfamide, carboplatin, etoposide (NICE) for patients with relapsed or refractory high-risk neuroblastoma: A single center, open-label phase 2 clinical trial. (ASCO 2025) - "11 NAX and 4 dinutuximab beta). Our clinical trial results suggest different chemotherapy combinations with naxitamab have the potential to rescue patients with R/R HR-NB. No unexpected toxicities were found when combining NAX with different chemotherapeutic agents."

Clinical • P2 data • Dermatology • Hematological Disorders • Immunology • Neuroblastoma • Oncology • Pain • Solid Tumor • Thrombocytopenia • Urticaria • CSF2

Dinutuximab beta for the treatment of Ewing sarcoma. (ASCO 2025) - "This study aims to investigate the efficacy of Dinutuximab beta in treating GD2-positive Ewing's sarcoma, both as a monotherapy and in combination with the standard chemotherapeutic agent doxorubicin. This pre-clinical study shows for the first time that dinutuximab beta in combination with standard of care improves survival. This lends support to continuing investigations of this novel therapeutic approach also in the clinical setting."

Ewing Sarcoma • Oncology • Sarcoma • Solid Tumor

Association of immunotherapy of high-risk neuroblastoma patients with long term infusion of dinutuximab beta with survival over short term infusion: Results from the HR-NBL1/SIOPEN trial. (ASCO 2025) - P3 | " High-risk patients as defined by metastatic disease (stage M) or local stage with MYC-N amplification received high intensity induction, surgery, high dose therapy with busulfan/melphalan followed by autologous stem cell transplantation (HDT/SCT) and local radiotherapy...All patients received 160 mg/m2 oral isotretinoin (d19-32)... We previously reported that LTI of DB increased the safety profile (less pain and inflammation) (Lancet Oncol 2018; 19(12):1617-1629; J Clin Oncol 37, 2019 (suppl; abstr 10013). Here we demonstrate that LTI is also associated with an improved outcome."

Clinical • Inflammation • Neuroblastoma • Oncology • Pain • Solid Tumor • IL2

Efficacy and Safety of Anti-GD2 Immunotherapy with Dinutuximab Beta in the Treatment of Relapsed/Refractory High-Risk Neuroblastoma. (PubMed, Target Oncol) - "Maintenance therapy for patients with R/R HR-NB with dinutuximab beta as monotherapy or in combination with isotretinoin demonstrated efficacy and acceptable safety. Further studies are needed in patients previously treated with anti-GD2 therapies to evaluate efficacy and impact on target antigens."

Journal • Review • Neuroblastoma • Oncology • Solid Tumor

Dinutuximab BetaVersus Naxitamab in the Treatment of Relapsed/Refractory Neuroblastoma - Matching-Adjusted Indirect Treatment Comparison and Economic Evaluation in Mexico (ISPOR 2025) - "Compared to naxitamab, dinutuximab beta improves survival in patients with relapsed/refractory neuroblastoma and is cost-saving in the health care system in Mexico."

HEOR • Neuroblastoma • Oncology • Solid Tumor • MYCN

Cost-Utility analysis of Dinutuximab Beta in Treating High-Risk Neuroblastoma: A Partitioned Survival Model Using R programming Language (ISPOR 2025) - "Dinutuximab beta is not a cost effective option compared to Isotretinoin at a threshold value of 40,000 JOD per QALY from a Jordanian health care system perspective. Price negotiations including customized risk sharing agreements are required to improve the value and affordability of Dinutuximab beta."

HEOR • Neuroblastoma • Oncology • Pediatrics • Solid Tumor

Assessment of Conditional Listing for Highly Uncertain and High-Priced Drugs in Taiwan (ISPOR 2025) - " A total of 12 drugs were considered for conditional listing between 2022 and 2024, which included Pemazyre, Tepmetko, Qarziba, Kymriah, Vitrakvi, Blincyto, Vydamax, Takhzyro, Polivy, Velexbru, Spevigo, and Koselugo. The conditional listing opens up new opportunities for high-priced drugs. However, the 2-year real-world evidence for Pemazyre remains insufficient for the government to make a final reimbursement decision. Consequently, the results of the HTA will pose challenges in the future in Taiwan."

Autophagy inhibition amplifies anti-tumor immunity effect of dinutuximab beta on neuroblastoma via the VEGFR/AKT/mTOR and ROS/NF-κB pathways. (PubMed, Int Immunopharmacol) - "Dinutuximab beta also triggers autophagy in NB cells, and inhibition of the VEGFR pathway with anlotinib amplifies dinutuximab beta-induced autophagy. Furthermore, autophagy inhibition by CQ enhances CXCL9 expression and anti-tumor T cell responses of single anti-GD2 therapy in vitro and in vivo. Collectively, this study suggests autophagy inhibitors may be a promising strategy for enhancing therapeutic efficacy in NB in conjunction with anti-GD2 immunotherapy."

IO biomarker • Journal • Neuroblastoma • Oncology • Solid Tumor • CD8 • CXCL9

Dinutuximab Beta Versus Naxitamab in the Treatment of Relapsed/ Refractory Neuroblastoma – Matching-Adjusted Indirect Treatment Comparison and Economic Evaluation in the Private Health Insurance System in Brazil (ISPOR 2025) - "Compared to naxitamab, dinutuximab beta improves survival in patients with relapsed/refractory neuroblastoma and is cost-saving in private health insurance system in Brazil."

HEOR • Reimbursement • US reimbursement • Neuroblastoma • Oncology • Solid Tumor • MYCN

LGND: 1Q:25 Results (Yahoo Finance) - "Qarziba (Recordati): Royalty revenue of $5.4 million recognized; Recordati will continue the geographic expansion of Qarziba; Qarziba may expand into Ewing sarcoma."

Sales • Ewing Sarcoma • Neuroblastoma • Neuroendocrine Tumor

Testing the Combination of Two Immunotherapy Drugs (Magrolimab and Dinutuximab) in Patients With Relapsed or Refractory Neuroblastoma or Relapsed Osteosarcoma (clinicaltrials.gov) - P1 | N=12 | Completed | Sponsor: National Cancer Institute (NCI) | Terminated ➔ Completed

Trial completion • Neuroblastoma • Oncology • Osteosarcoma • Sarcoma • Solid Tumor

Isotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma (clinicaltrials.gov) - P3 | N=1449 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Mar 2025 ➔ Mar 2026

Trial completion date • Bone Marrow Transplantation • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor • Transplantation

Irinotecan Hydrochloride, Temozolomide, and Dinutuximab With or Without Eflornithine in Treating Patients With Relapsed or Refractory Neuroblastoma (clinicaltrials.gov) - P2 | N=94 | Active, not recruiting | Sponsor: Children's Oncology Group | Trial completion date: Mar 2025 ➔ Apr 2026

Trial completion date • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor

TACKLING THE PROBLEM OF FALSE-NEGATIVE RESULTS OF CD34+ CELL ENUMERATION IN PEDIATRIC NEUROBLASTOMA PATIENTS PLANNED FOR STEM CELL COLLECTION: INSIGHTS FROM TWO CASES (EBMT 2025) - " Case 1: A 3-year-old boy with high-risk neuroblastoma, in first complete remission (CR1) after induction chemotherapy followed by immunotherapy with anti-GD2 antibody dinutuximab beta, was scheduled for SC collection after mobilization with granulocyte colony-stimulating factor (G-CSF) and plerixafor. The described cases highlight the critical need for awareness among medical and laboratory professionals regarding potential false-negative results of CD34+ cell enumeration, that could be related to interference, cross-reactivity or high titers of antibodies, either autoimmune or drug-induced, known as a hook effect. Sample dilution serves as a simple yet efficient solution to this diagnostic pitfall, facilitating timely and accurate SC collection, thus preventing patient exposure to unnecessary procedures and treatment delays. Further research is warranted to determine the prevalence and mechanisms of this phenomenon."

Clinical • Bone Marrow Transplantation • CNS Tumor • Immunology • Neuroblastoma • Oncology • Pediatrics • Solid Tumor • CD34

NANT: Lenalidomide and Dinutuximab With or Without Isotretinoin in Treating Younger Patients With Refractory or Recurrent Neuroblastoma (clinicaltrials.gov) - P1 | N=27 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Mar 2025 ➔ Mar 2026

Trial completion date • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor

A clinical observational study of dinutuximab beta as first-line maintenance treatment for patients with high-risk neuroblastoma in China. (PubMed, BMC Pediatr) - "The study showed promising efficacy and safety of dinutuximab beta as the first-line maintenance immunotherapy for pediatric patients with HR-NB. Notably, the combination of dinutuximab beta with GM-CSF and VIT chemotherapy could be used for treating patients who did not achieve CR after previous multimodal therapy."

Journal • Observational data • Retrospective data • CNS Tumor • Neuroblastoma • Oncology • Pediatrics • Solid Tumor • CSF2

3-yr-old given life-saving immunotherapy for cancer (The Times of India) - "A three-year-old girl diagnosed with high-risk neuroblastoma, a type of cancer, received life-saving immunotherapy called Dinutuximab beta at Kasturba Hospital, Manipal. The only treatment that could cure her of this cancer was immunotherapy, stated the hospital spokesperson....This is the first patient to undergo a successful immunotherapy course here."

Clinical • Neuroblastoma

International Phase I Trial of Dinutuximab Beta With VDC/IE in GD2-Positive Ewing Sarkoma (clinicaltrials.gov) - P1 | N=18 | Not yet recruiting | Sponsor: Prof. Dr. Dirk Reinhardt

New P1 trial • Ewing Sarcoma • Oncology • Pediatrics • Sarcoma • Solid Tumor

Buoyed by Qarziba’s success, MFDS expands support for innovative drugs (Korea Biomedical Review) - "At a news conference on Tuesday, the National Institute of Food and Drug Safety Evaluation (NIFDS) under the ministry unveiled plans to strengthen support for developing innovative products, including new drugs, advanced biopharmaceuticals, and innovative medical devices....The support expansion is based on the rapid approval and reimbursement of rare disease treatments, including Qarziba (dinutuximab beta), a neuroblastoma drug....As a result, Qarziba, which was selected for the first pilot project, was covered by insurance within six months of its approval last year."

Reimbursement • Neuroblastoma

PHOX2B-associated Congenital Central Hypoventilation Syndrome Revealed Upon Treatment With Dinutuximab-beta. (PubMed, J Pediatr Hematol Oncol) - "CCHS was revealed upon severe respiratory decompensation while the patient was administered the anti-GD2 antibody dinutuximab-beta, as part of neuroblastoma treatment. From this experience, we make propositions for the management of patients with high-risk neuroblastoma and a constitutional pathogenic variant of PHOX2B."

Journal • Brain Cancer • CNS Tumor • Neuroblastoma • Oncology • Respiratory Diseases • Solid Tumor • HOXB6 • PHOX2B

Strategies to manage the adverse effects of immunotherapy with dinutuximab beta in neuroblastoma: an Italian experience and literature review. (PubMed, Support Care Cancer) - "In the experience of the authors, adverse events associated with dinutuximab beta may be prevented and managed in experienced centers. The supportive therapy may be reduced after the first cycle to improve the quality of life."

Adverse events • Journal • Review • Allergy • CNS Tumor • Neuralgia • Neuroblastoma • Oncology • Pain • Pediatrics • Solid Tumor

DiTuSarc: Treatment With Dinutuximab Beta, Zoledronic Acid and Low-dose Interleukin (IL-2) in Patients With Leiomyosarcoma (clinicaltrials.gov) - P2 | N=7 | Completed | Sponsor: Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest | Active, not recruiting ➔ Completed | N=10 ➔ 7 | Trial primary completion date: Aug 2024 ➔ Jan 2025

Enrollment change • Trial completion • Trial primary completion date • Leiomyosarcoma • Oncology • Sarcoma • Solid Tumor

131I-mIBG THERAPY IN RELAPSED/REFRACTORY NEUROBLASTOMA: A WEAPON FROM THE FUTURE PAST. (PubMed, Crit Rev Oncol Hematol) - "Neuroblastoma (NB) is the most common extracranial solid tumor in children, with variable outcomes ranging from spontaneous remission to high-risk cases often leading to relapse or refractory disease. Our systematic review, based on MEDLINE, EMBASE, and Cochrane CENTRAL databases up to December 2023, evaluates the effectiveness and toxicity of ¹³¹I-mIBG therapy in relapsed/refractory NB. It also discusses its potential role in conjunction with emerging therapies like CAR-T cells, haploidentical stem cell transplantation, and dinutuximab beta."

IO biomarker • Journal • Review • Bone Marrow Transplantation • CNS Tumor • Neuroblastoma • Oncology • Pediatrics • Solid Tumor • Transplantation

Inbraced: Phase I Study of 131-I MIBG Followed by Nivolumab & Dinutuximab Beta Antibodies in Children with Relapsed/refractory Neuroblastoma (clinicaltrials.gov) - P1 | N=44 | Active, not recruiting | Sponsor: University Hospital Southampton NHS Foundation Trust | Recruiting ➔ Active, not recruiting | Trial primary completion date: Sep 2024 ➔ May 2025

Enrollment closed • Trial primary completion date • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor