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. Author manuscript; available in PMC: 2021 Mar 15.
Published in final edited form as: Cancer. 2019 Dec 3;126(6):1315–1321. doi: 10.1002/cncr.32649

Table 1:

Single agent targeted therapies approved for genomically altered cancers (mutation, amplification or fusion) and response rates*

Solid tumors: Fusion target
Drug Disease Indication Line of Therapy Aberrant gene Number of studies** Response Rate (%)**
Alectinib NSCLC 2+ ALK 1 79%
Brigatinib NSCLC 2+ ALK 1 53%
Ceritinib NSCLC 2+ ALK 1 73%
Crizotinib NSCLC 1 ALK/ROS1 2 56%
Entrectinib Solid tumors 1+ NTRK/ROS1/ALK 1 78%
Larotrectinib Solid tumors 1+ NTRK 1 75%
Solid tumor: Non-fusion target
Drug Disease Indication Line of Therapy Aberrant gene Number of studies** Response Rate (%)
Afatinib NSCLC 1+ EGFR 1 50%
Dabrafenib Melanoma 1+ BRAF 1 54%
Dacomitinib NSCLC 1+ EGFR 1 75%
Erlotinib NSCLC 1+ EGFR 1 65%
Gefitinib NSCLC 1 EGFR 1 70%
Imatinib GIST 1 KIT 1 51%
Olaparib Ovarian 4+ BRCA 1 34%
Osimertinib NSCLC 1+ EGFR 1 51%
Rucaparib Ovarian 3+ BRCA 1 54%
Talazoparib Breast 2+ BRCA 1 50%
TDM-1 Breast 2+ HER2 1 44%
Trastuzumab Breast 2+ HER2 1 14%
Vemurafenib Melanoma 1+ BRAF 1 48%
Hematologic malignancies: Fusion Target
Drug Disease Indication Line of Therapy Aberrant gene Number of studies** Response Rate (%)
All trans-retinoic acid APL 1+ PML-RARA 1 72%
Bosutinib CML 2+ BCR-ABL 2 31%
Dasatinib CML 1+ BCR-ABL 2 63%
Imatinib CML 1 BCR-ABL 1 73%
Nilotinib CML 1+ BCR-ABL 1 84%
Ponatinib CML 2+ BCR-ABL 1 46%
Hematologic malignancies: Target non-fusion
Drug Disease Indication Line of Therapy Aberrant gene Number of studies** Response Rate (%)
Enasidenib AML 2+ IDH2 1 23%
Gilteritinib AML 2+ FLT3 1 21%
Ivosidenib AML 2+ IDH1 1 33%
Vemurafenib ECD 1+ BRAF 1 55%
*

Approved by the Food and Drug Administration (FDA)

**

Refers to number of studies provided in FDA package insert; Response rate is per package insert. If more than one study listed, the mean is given

Abbreviations: AML=acute myelogenous leukemia; CML=chronic myelogenous leukemia; ECD=Erdheim-Chester disease; NHL=non-Hodgkin’s lymphoma; NSCLC=non-small cell lung cancer