Regulatory approval
Published by the Food and Drug Administration.
The U.S. Food and Drug Administration granted accelerated approval to pembrolizumab for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [>=10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. The product label states that the safety and effectiveness of pembrolizumab in pediatric patients with TMB-H central nervous system cancers have not been established and that this indication is approved under accelerated approval based on tumor response rate and durability of response. Furthermore, it states that continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
This is written in the approval document as:
KEYTRUDA is a programmed death receptor-1 (PD-1)-blocking antibody indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [>=10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Citation
Therapeutic response
Precision oncology relationships for therapeutic response derived from this regulatory approval.
| Type | Biomarker(s) | Cancer type | Therapy(ies) | |
|---|---|---|---|---|
| Sensitivity (+) | TMB-H (>= 10 mutations / Mb) | Any solid tumor | Pembrolizumab |