Implementation status of pharmacological studies in the development of orphan drugs

Table 2 Vemurafenib development for malignant melanoma with mutations in specific amino acids

BRAF	in vitro			In vivo	Clinical trial (Phase III)
BRAF	Kinase inhibition	Inhibition of cell phosphorylation	Growth inhibition		Inclusion criteria	Vemurafenib (n = 336)	Control (n = 337)
V600E mutation	〇	〇	〇	〇	〇	295(88%)	303(90%)
V600D mutation	〇	〇	〇		△^a	0	1
V600K mutation	〇				△^a	33(10%)	24(7%)
Other mutations	10 other V600 mutations	V600R mutation				0	1
Wild type	〇	〇	〇			1	1

Mutated cell lines used in non-clinical studies of cancer drugs with mutations in specific amino acids and inclusion criteria for clinical trials
Growth inhibitory effects were evaluated in vitro using cancer cells with target mutations
^aThe Cobas® BRAF V600 mutation test shows cross-reactivity to BRAF V600K and V600D

ISSN: 1750-1172