Pediatric Oncology

Tisagenlecleucel is moving to earlier lines of therapy for pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia.

Opaganib has received orphan drug designation from the FDA for use in patients with neuroblastoma.

The FDA has awarded rare pediatric disease designation and orphan drug designation to INV724 for the treatment of neuroblastoma.

Luis E. Raez, MD, FACP, FCCP, FASCO, discusses the FDA approval of repotrectinib for NTRK-positive advanced solid tumors.

The FDA has approved vorasidenib for grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation.

In case you missed any, below is a recap of every OncLive On Air episode that aired in July 2024.

The FDA has accepted the resubmission of a BLA for remestemcel-L in pediatric steroid-refractory acute graft-vs-host-disease.

The BLA seeking approval for remestemcel-L for children with steroid-refractory acute graft-vs-host-disease has been resubmitted to the FDA.

Daniel M. Halperin, MD, details the FDA approval of lutetium Lu 177 dotatate as well as safety and efficacy data in pediatric and adult patients treated with the agent.

The FDA has granted accelerated approval to selpercatinib for pediatric patients with select RET-altered metastatic thyroid cancers or solid tumors.

The FDA granted accelerated approval to tovorafenib for pediatric patients relapsed/refractory low-grade glioma with a BRAF fusion/rearrangement or a BRAF V600 mutation.

The FDA has approved lutetium Lu 177 dotatate for pediatric patients 12 years of age and older with SSTR–positive GEP-NETs.

A unique approach combining genomic and sensitivity testing with machine learning was feasible for guiding treatment selection in pediatric cancers.

In case you missed it, these were the key regulatory decisions made by the FDA in March 2024.

The FDA has approved inotuzumab ozogamicin for pediatric patients with relapsed/refractory CD22-positive B-cell precursor acute lymphoblastic leukemia.

Burton Eliot Appel, MD, discusses the importance of recognizing Childhood Cancer Day in pediatric patients with cancer.

Burton Eliot Appel, MD, discusses the landscape of pediatric oncology on International Childhood Cancer Day.

The FDA has approved eflornithine (Iwilfin) as a treatment to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma who have achieved at least a partial response to prior multi-agent, multimodality therapy including anti-GD2 immunotherapy.

The FDA has granted priority review to a new drug application seeking the approval of tovorafenib monotherapy for the treatment of pediatric patients with relapsed or progressive low-grade glioma.

The FDA has granted accelerated approval to entrectinib (Rozlytrek) for pediatric patients aged older than 1 month with solid tumors that harbor a NTRK gene fusion without a known acquired resistance mutation, are metastatic, or where surgical resection is likely to result in severe morbidity, and have progressed after treatment or have no satisfactory standard therapy options.

The FDA has approved nivolumab (Opdivo) for the adjuvant treatment of adult and pediatric patients 12 years of age and older with completely resected stage IIB or IIC melanoma.

In a 14-to-6 vote, the FDA’s Oncologic Drugs Advisory Committee voted sufficient evidence has been provided to demonstrate the event-free survival benefit of eflornithine to reduce the risk of relapse in pediatric patients with high-risk neuroblastoma.

The FDA has approved bosutinib (Bosulif) for the treatment of pediatric patients 1 year of age and older with Philadelphia chromosome–positive, chronic-phase chronic myelogenous leukemia that is newly diagnosed or resistant or intolerant to prior therapy.

The European Commission has granted marketing authorization for Enrylaze for use as a component of a multi-agent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia and lymphoblastic lymphoma in adult and pediatric patients 1 month and older who developed hypersensitivity or silent inactivation to E. coli–derived asparaginase.

A rolling new drug application seeking the approval of tovorafenib monotherapy for the treatment of patients with relapsed or progressive pediatric low-grade glioma has been submitted to the FDA.

The FDA has placed a clinical hold on the phase 1 PLAT-08 trial evaluating SC-DARIC33 in pediatric and young-adult patients with relapsed/refractory CD33-positive acute myeloid leukemia.

Alex Herrera, MD, discussed key data from the SWOG S1826 trial and other important research in hematologic malignancies presented at the 2023 ASCO Annual Meeting.

The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended the approval of JZP458 for use in combination with multi-agent chemotherapy for the treatment of adult and pediatric patients 1 month and older with acute lymphoblastic leukemia and lymphoblastic lymphoma who developed hypersensitivity or silent inactivation to Escherichia coli–derived asparaginase.

The phase 1 PLAT-08 trial investigating SC-DARIC33 in pediatric and young-adult patients with relapsed/refractory CD33-positive acute myeloid leukemia has been paused following the report of a grade 5 serious adverse effect.

The investigational CAR T-cell therapy GD2-CART01 showed preliminary antitumor activity and safety in pediatric patients with relapsed or refractory high-risk neuroblastoma, according to data from a phase 1/2 trial.