13
Table 2. Selected Genetic Alterations Linked to FDA
Approvals as of June 2021
a
Genetic Alterations Tumor type Targeted Therapeutics
FDA-approved treatments for specific genetic alterations in specific tumor types (cont'd)
PDGFRA exon 18
mutations
Gastrointestinal stromal
tumor
Avapritinib
Oncogenic mutations
in PIK3CA
HR+ HER2-breast
cancer
Fulvestrant + Alpelisib
RET fusions NSCLC, thyroid cancer Pralsetinib, Selpercatinib
Oncogenic mutations
in RET
Medullary thyroid cancer Pralsetinib, Selpercatinib
ROS1 fusions NSCLC Crizotinib, Entrectinib
FDA-approved treatments for specific biomarkers in tumor type agnostic indications
NTRK1 and/or
NTRK2 and/or
NTRK3 fusions
Solid tumors Entrectinib, Larotrectinib
MSI-H, tumor
mutational burden-high
(TMB-H)
Solid tumors Pembrolizumab
FDA-approved treatments that are not biomarker-linked in solid tumors characterized
by specified genetic alterations
Oncogenic mutations
in NF1
Neurofibroma Selumetinib
COL1A1-PDGFB
fusions
Dermatofibrosarcoma
protuberans
Imatinib
SMARCB1 deletions Epithelioid sarcoma Tazemetostat
Oncogenic mutations
in TSC1 and TSC2
Subependymal giant-cell
astrocytomas (SEGA)
Everolimus
KIT exon 11, 9, 13, 14
and 17 mutations
Gastrointestinal stromal
tumor
Imatinib, Sunitinib (post-
progression on imatinib),
Regorafenib (post-progression
on imatinib and sunitinib),
Ripretinib (post-progression on
≥3 kinase inhibitors including
imatinib)
(cont'd)
