On March 14, the Food and Drug Administration (FDA) approved pembrolizumab (Keytruda®) for the treatment of some patients with classical Hodgkin lymphoma (cHL). The drug is approved for the treatment of both adult and pediatric patients with refractory cHL, or patients whose disease has relapsed after three or more lines of therapy.
The FDA’s action makes pembrolizumab the first immune checkpoint inhibitor to be approved for the treatment of children with cancer. The drug has previously been approved for the treatment of adult patients with head and neck cancer, melanoma, and lung cancer.
Pembrolizumab received accelerated approval for the new indication based on data from a single-arm clinical trial with more than 200 adult patients. All patients in the trial had cHL that had returned after multiple prior treatments or was no longer responding to treatments that include an autologous stem cell transplantation and/or brentuximab vedotin (Adcetris®). With an accelerated approval, the FDA requires that the drug’s manufacturer conduct further studies to confirm the clinical benefit for the indication.
No pediatric patients participated in the clinical trial on which the approval was based. In this instance, the FDA extrapolated the efficacy in pediatric patients from the results that were observed in adults.