Table 1.

Main Clinical Trials Leading to Other Indications FDA Approved for Pembrolizumab

Disease Site	Study	n	Major Efficacy Outcome Measures	ORR	Median Response Duration (Months)	Median PFS (Months)	HR (95% CI)	Median OS (Months)	HR (95% CI)	FDA Approval
cHL	KEYNOTE-087144	210	ORR, Complete Response Rate, and DoR	69% (95% CI: 62, 75)	11.1 (0+, 11.1) (for the 145 patients)	–	–	–	-	03/2017
Gastrointestinal cancers	KEYNOTE-0591,145,146	259	ORR and DoR	13.3% (95% CI: 8.2, 20.0) (for the 143 patients)	8.4 (1.6+, 17.3+)	2.0 (95% CI: 2.0, 2.1)	–	5.6 (95% CI: 4.3, 6.9)	–	09/2017
Cervical cancer	KEYNOTE-158147	98	ORR and DoR	14.3% (95% CI: 7.4, 24.1) (for the 77 patients)	–	2.1	–	9.4	–	06/2018
PMBCL	KEYNOTE-170148	53	ORR and DoR	45% (95% CI: 32, 60)	NR (1.1+, 19.2+) (for the 24 patients)	–	–	–	–	06/2018
HCC	KEYNOTE-224149	104	ORR and DoR	17% (95% CI: 11, 26)	NR (3.1+, 14.6+)	4.9 (95% CI: 3.4, 7.2)	–	12.9 (95% CI: 9.7, 15.5)	–	11/2018
MCC	KEYNOTE-017150	50	ORR and DoR	56% (95% CI: 41, 70)	NR (5.9, 34.5+)	16.8 (95% CI: 4.6, not estimable)	–	NR (95% CI: 26.0, not estimable)	–	12/2018
RCC	KEYNOTE-426151	861	OS and PFS	59% (95% CI: 54, 64)	–	15.1 (95% CI: 12.6, 17.7)	0.69 (0.56, 0.84)	NR (NR, NR)	0.53 (0.38, 0.74)	04/2019
Esophageal squamous cell cancer	KEYNOTE-181152	628	OS	22% (95% CI: 14, 33)	9.3 (2.1+, 18.8+)	3.2 (95% CI: 2.1, 4.4)	0.66 (0.48, 0.92)	10.3 (95% CI: 7.0, 13.5)	0.64 (0.46, 0.90)	07/2019
	KEYNOTE-180153	121	ORR and DoR	20% (95% CI: 8, 37) (for the 35 patients)	–	–	–	–	–
Endometrial carcinoma	KEYNOTE-146154	108	ORR and DOR	38.3% (95% CI, 29, 49)	NR (1.2+, 33.1+) (for the 36 patients)	–	–	–	–	09/2019
NMIBC	KEYNOTE-057155	148	Complete Response and DoR	-	16.2 (0.0+, 30.4+)	–	–	–	–	01/2020