First Pediatric Approval for Brentuximab Vedotin 

The CD30-targeted antibody-drug conjugate was approved for some children with blood cancer. 

The U.S. Food and Drug Administration (FDA) has approved brentuximab vedotin (Adcetris) in combination with certain cancer therapies for pediatric patients over the age of 2 with untreated, high-risk classical Hodgkin lymphoma (cHL). This marks the first pediatric approval for brentuximab vedotin, which was previously approved for adult patients with certain lymphomas. 

Brentuximab vedotin is a type of targeted therapy known as antibody-drug conjugate. It consists of an antibody that directs it to deliver a cell-killing drug to cells that express the CD30 protein on their surface. CD30 expression is characteristic of Hodgkin lymphomas, so brentuximab vedotin selectively targets these cancer cells.  

The approval is based on results from the randomized, open-label, actively controlled phase III AHOD1331 clinical trial. The trial enrolled 600 pediatric patients (median age of 15 years) with high-risk cHL (defined as stage IIB with bulk disease, stage IIIB, stage IVA, or stage IVB). Patients were randomly assigned to receive either brentuximab vedotin plus the drugs doxorubicin, vincristine, etoposide, prednisone, and cyclophosphamide; or a combination of doxorubicin, bleomycin, vincristine, etoposide, prednisone, and cyclophosphamide. 

Patients in the brentuximab vedotin arm were 59 percent less likely than those in the control arm to experience disease progression, relapse, secondary malignancy, or death. Eight percent of patients in the brentuximab vedotin arm experienced these events, as opposed to 17 percent of patients in the control arm.  

The approval is accompanied by a boxed warning highlighting the risk of progressive multifocal leukoencephalopathy and death in patients receiving brentuximab vedotin. 

cHL is a blood cancer that accounts for approximately 6 percent of all childhood cancers. The disease is most common in adolescents. The five-year relative survival of pediatric cHL is 98 percent. 

The FDA rendered its decision on November 10, 2022.