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

Dinutuximab beta for the treatment of Ewing sarcoma. (ASCO 2025) - "The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Ewing Sarcoma • Oncology • 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

Assessment of Conditional Listing for Highly Uncertain and High-Priced Drugs in Taiwan (ISPOR 2025) - "The review and assessment process were conducted independently by two researchers. 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."

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 | "Clinical Trial Registration Number: 2006-001489-17 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Clinical • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor

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 • CNS Tumor • Neuroblastoma • Oncology • Pediatrics • 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 • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor • MYCN

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 • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor • MYCN

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

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 | Terminated | Sponsor: National Cancer Institute (NCI) | Trial completion date: Nov 2025 ➔ Sep 2024 | Active, not recruiting ➔ Terminated; Drug supply issues

Trial completion date • Trial termination • CNS Tumor • Neuroblastoma • Oncology • Osteosarcoma • Sarcoma • Solid Tumor

Chemoimmunotherapy With Dinutuximab Beta Compared to Chemotherapy Regimens in Patients With Relapsed/Refractory Neuroblastoma: Systematic Literature Review and Indirect Treatment Comparison (ISPOR-EU 2024) - "Prospective and retrospective studies published or presented at conferences from 2000 in patients treated with temozolomide plus irinotecan (TEMIRI), temozolomide plus topotecan (TOTEM), cyclophosphamide plus topotecan, with and without dinutuximab beta and with and without bevacizumab, were included. Our results demonstrate improved objective response and progression free survival outcomes with dinutuximab beta chemoimmunotherapy in relapsed/refractory neuroblastoma and support recommendations for its use in this setting. Analysis with individual patient data adjusted for predictors of outcomes would allow for a more precise estimate of treatment effect."

Clinical • Review • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor

NPPA asks companies to revise MRP of three anti-cancer drugs and submit information (Pharmabiz) - "The National Pharmaceutical Pricing Authority (NPPA) has directed all manufacturers and marketing companies selling the three anti-cancer drugs that were exempted from customs duty and Goods and Services Tax (GST) earlier this year, to revise the maximum retail price (MRP) of these drugs and submit the details regarding the revision with the regulators...The move is in line with the Centre's announcement in the Union Budget 2024-25 to fully exempt cancer drugs namely, trastuzumab deruxtecan, osimertinib and durvalumab from customs duty....It may be noted that the GST Council in its 50th meeting has recommended IGST exemption of dinutuximab (Qarziba), another cancer drug, when imported for personal use."

Commercial • Biliary Tract Cancer • Breast Cancer • Gastric Cancer • Gastrointestinal Cancer • HER2 Breast Cancer • HER2 Positive Breast Cancer • Lung Cancer • Neuroblastoma • Non Small Cell Lung Cancer • Oncology • Solid Tumor

Iwilfin (eflornithine) approved by the FDA as the first and only oral maintenance therapy for high-risk neuroblastoma in adult and pediatric patients: Narrative review. (PubMed, Medicine (Baltimore)) - "While dinutuximab beta immunotherapy is an effective and widely used treatment, it has several potential side effects that must be carefully monitored...However, DFMO has adverse effects that require continuous monitoring. Further research is needed to minimize these side effects and improve its efficacy, particularly in addressing resistance caused by long-term use."

FDA event • IO biomarker • Journal • Review • CNS Tumor • Neuroblastoma • Oncology • Pediatrics • Solid Tumor • MYCN

Immunotherapy Qarziba to be reimbursed for high-risk neuroblastoma patients starting next month (Korea Biomedical Review) - "Starting next month, the immunotherapy drug Qarziba (dinutuximab beta), a critical treatment option for high-risk and relapsed/refractory neuroblastoma patients, will be covered by health insurance in Korea. According to Recordati Korea, Qarziba will be reimbursed for pediatric neuroblastoma patients starting next month, as announced by the Ministry of Health and Welfare. It is currently the only licensed immunotherapy in Korea for high-risk and relapsed/refractory neuroblastoma. The Ministry of Health and Welfare specified that Qarziba will be covered for neuroblastoma patients aged 12 months to 20 years who have not received anti-GD2 antibody treatment before and meet specific criteria....The reimbursement decision was supported by pivotal studies, including the phase 3 APN311-302 trial."

Reimbursement • Neuroblastoma

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 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | N=82 ➔ 12 | Trial completion date: Sep 2024 ➔ Nov 2025

Enrollment change • Trial completion date • CNS Tumor • Neuroblastoma • Oncology • Osteosarcoma • Sarcoma • Solid Tumor