Targeted therapies are designed to attack or kill cancer cells, while sparing normal cells as much as possible. These therapies are often designed to attach to abnormal proteins, receptors or genes that are found in high quantities in cancer cells or the surrounding tissue.
Some, but not all targeted therapies work best against cancer cells with certain markers or mutations. Two important types of tests can help guide selection of these targeted therapies.
testing involves looking at tumor, blood or other other tissue samples for abnormal markers that might indicate the cancer is likely to respond to a particular targeted therapy. You can learn more about biomarker testing for selecting targeted therapies in our Biomarkers sections.
Genetic testing for inherited mutations can also help guide treatment with targeted therapies. PARP inhibitors, for example, are a type of targeted therapy that are most effective for treating cancer in people with a or mutation.
Each targeted therapy drug has different indications. Like all cancer treatments, targeted therapies can have side effects. Visit our section on Side Effects for more information.
PARP inhibitors
PARP inhibitors work by blocking a protein used to repair damaged . They were initially developed to treat cancers in people with an inherited BRCA1 or BRCA2 mutation. Since then, research and additional approvals have expanded use of PARP inhibitors to more people and situations. There are five PARP inhibitors that have received FDA approval for treating different types of cancers.
Indications vary by:
type and of cancer: PARP inhibitors have been approved to treat breast, ovarian, pancreatic and cancers.
presence of a mutation or biomarker: PARP inhibitors have been approved in different settings for:
people with certain inherited mutations.
people with certain acquired mutations.
people with certain tumor biomarkers.
women with certain types of ovarian cancer, regardless of the presence of an or biomarker.
number of and response to prior treatments: have been approved in different settings for:
platinum-sensitive or partially platinum-sensitive ovarian cancer.
castration-resistent prostate cancer (mCRPC).
after chemotherapy.
to treat progression or recurrence after a specific number of prior treatments.
Clinical trials studying PARP inhibitors in new settings or combinations are enrolling patients. As research continues, these approvals may expand to include treatment for additional cancers, earlier stages of cancer, people with other inherited mutations, and based on different tumor biomarkers.
Table of PARP inhibitors
Table of PARP Inhibitors: This table lists different PARP inhibitors and their indications.
Drug
Cancer Type
Stage
Use
Biomarker
Lynparza ()
Breast cancer
Early stage breast cancer at high risk for recurrence
Given for one year as after completion of or neoadjuvant chemotherapy and local treatment (surgery and, or radiation).
BRCA1 or BRCA2 inherited mutation ()
Lynparza (olaparib)
Breast cancer
Metastatic
For treatment of patients who have previously received chemotherapy, or hormone therapy for patients with hormone receptor disease.
BRCA1 or BRCA2 inherited mutation and
()
Breast cancer
Metastatic
For treatment of metastatic breast cancer.
BRCA1 or BRCA2 mutation and HER2-negative
Lynparza (olaparib)
Ovarian cancer
Stage 3 or 4
maintenance therapy for people who had a complete or partial response to platinum chemotherapy.
Inherited () mutation in BRCA1 or BRCA2
Tumor (somatic) mutation in BRCA1 or BRCA2
Lynparza (olaparib)
Ovarian cancer
Stage 3 or 4
Combined with Avastin (bevacuzimab) for people who had a complete or partial response to platinum chemotherapy.
Inherited mutation in BRCA1 or BRCA2, or
HRD-positive ( Deficiency-positive)
Lynparza (olaparib)
()
()
Ovarian cancer
Stage 3 or 4
Second-line or later maintenance therapy for people who had a complete or partial response to platinum chemotherapy.
No biomarker needed
Zejula (niraparib)
Ovarian cancer
Stage 3 or 4
For people who had a complete or partial response to platinum chemotherapy.
No biomarker required
Lynparza (olaparib)
Pancreatic cancer
Metastatic
For people whose disease has not progressed on at least 16 weeks of platinum-based chemotherapy.
Inherited mutation in BRCA1 or BRCA2
Akeega (niraparib and acetate)
Prostate cancer
Metastatic castration-resistant prostate cancer (mCRPC)
In combination with prednisone for first-line or later treatment of mCRPC.
Inherited or tumor mutation in BRCA1 or BRCA2 based on FoundationOne tumor test
Lynparza (olaparib)
Prostate cancer
Metastatic castration-resistant prostate cancer (mCRPC)
Combined with Zytiga and prednisone or prednisolone for first-line or later treatment of mCRPC.
Inherited mutation in BRCA1 or BRCA2 found through genetic testing, or
Tumor BRCA1 or BRCA2 mutation found through tumor testing or
Lynparza (olaparib)
Prostate cancer
Metastatic castration-resistant prostate cancer (mCRPC)
For treatment of mCRPC which has progressed following treatment with Xtandi () or Zytiga (abiraterone).
Inherited mutation in BRCA1 or BRCA2, or
Tumor mutation in one of the following genes: , BRCA1, BRCA2, , , CDK12, , FANCL, , RAD51B, , , RAD54
Rubraca (rucaparib)
Prostate cancer
Metastatic castration-resistant prostate cancer (mCRPC)
For treatment of mCRPC which has been treated with androgen receptor-directed therapy and a taxane-based chemotherapy.
Inherited mutation in BRCA1 or BRCA2 found through genetic testing, or
Tumor BRCA1 or BRCA2 mutation found through tumor testing or liquid biopsy
Talzenna (talazoparib)
Prostate cancer
Metastatic castration-resistant prostate cancer (mCRPC)
In combination with enzalutamide for mCRPC which has not yet been treated in the castration-resistant setting.
Inherited or tumor mutation in one of the following genes: BRCA1, BRCA2, ATM, ATR, CDK12, CHEK2, FANCA, , MRE11A, , PALB2, or RAD51C
Other targeted therapies
There is a growing list of FDA-approved targeted therapies for cancer treatment. Most are classified by the abnormal target that they are designed to bind to and attack. Many of these new therapies are approved for use in all types of cancers () as long as the cancer contains the right marker or target. Drugs that are approved across cancer types are sometimes called pan-tumor or tumor-agnostic therapies.
Some drugs are classified as both immunotherapies and targeted therapies because they use antibodies to target abnormal proteins or receptors that are found in high quantities in cancer cells or the surrounding tissue. Antibody drug conjugates (ADCs) are drugs that combine two different types of molecules. A chemotherapy drug is linked to an antibody that delivers the chemotherapy directly to the cancer cells.
The table below lists some common targeted therapies used in cancer treatment. Clincal trials are studying new agents for treating cancer.
Table of targeted therapies
Table of Targeted Therapies: This table lists commonly used targeted therapies and their indications.
Drug
Cancer Type
Stage
Use
Biomarker
Type of Agent
Afinitor (everolimus)
Breast cancer
Metastatic or advanced
Combined with exemestane for postmenopausal women with advanced breast cancer which progressed with letrozole or anastrozole.
HR-positive, HER2-negative
MTOR inhibitor
Afinitor (everolimus)
Pancreatic neuro-endocrine tumors (PNET)
Progressive PNETs
Used to treat PNETs that have progressed on other treatments.
No biomarker needed
MTOR inhibitor
Avastin (bevacizumab)
Ovarian, or primary peritoneal cancer
Stage 2-4
Combined with Lynparza (olaparib) for first-line maintenance therapy for platinum-sensitive cancer.
() testing
VEGF inhibitor
Avastin (bevacizumab)
Ovarian, fallopian tube or primary peritoneal cancer
Stage 3-4
Combined with chemotherapy, followed by Avastin as a single agent following initial surgical resection.
No biomarker needed
VEGF inhibitor
Avastin (bevacizumab)
Ovarian, fallopian tube or primary peritoneal cancer
Recurrent
Combined with chemotherapy for treating platinum-resistant, recurrent disease in people who received no more than 2 prior chemotherapy regimens.
No biomarker needed
VEGF inhibitor
Avastin (bevacizumab)
Ovarian, fallopian tube or primary peritoneal cancer
Recurrent
Combined with chemotherapy, followed by Avastin as a single agent, for platinum-sensitive recurrent disease.
No biomarker needed
VEGF inhibitor
Avastin (bevacizumab)
Colorectal cancer
Metastatic
Combined with intravenous 5-fluorouracil-based chemotherapy for first-, or second-line treatment.
No biomarker needed
VEGF inhibitor
Avastin (bevacizumab)
Colorectal cancer
Metastatic
Combined with chemotherapy for second-line treatment in patients who have progressed on a first-line Avastin-containing regimen.
No biomarker needed
VEGF inhibitor
Braftovi (encorafenib)
Colorectal cancer
Metastatic
Combined with cetuximab, for the treatment of adult patients with metastatic colorectal cancer.
BRAF V600E tumor mutation
BRAF inhibitor
Braftovi (encorafenib)
Melanoma
Metastatic
Combined with Mektovi (binimetinib), for the treatment of patients with unresectable or metastatic melanoma.
BRAF V600E or V600K tumor mutation
BRAF inhibitor
Cotellic (cobimetinib)
Melanoma
Metastatic
Combined with Zelboraf (vemurafenib) for the treatment of patients with unresectable or metastatic melanoma.
BRAF V600E or V600K tumor mutation
BRAF inhibitor
Cyramza (ramucirumab)
Colorectal cancer
Metastatic
Combined with FOLFIRI chemotherapy, for treatment after disease progression on, or after prior therapy with bevacizumab, oxaliplatin, and a fluoropyrimidine.
No biomarker required
VEGF inhibitor
Datroway (datopotamab deruxtecan-dlnk)
Breast cancer
Metastatic
Treatment for cancers that have progressed after hormone therapy and chemotherapy.
Hormone receptor position (HR-positive), HER2-negative
Antibody-drug conjugate (chemotherapy attached to antibody targeting TROP-2)
ELAHERE (mirvetuximab soravtansine-gynx)
Ovarian cancer
Stage 3-4
Used as second-line or later treatment of platinum-resistant or platinum-sensitive recurrent ovarian cancer.
Positive for FRα (folate receptor alpha)
Antibody-drug conjugate (chemotherapy attached to antibody targeting FR-α receptor)
Enhertu (fam-trastuzumab-deruxtecan-nxki)
Breast cancer
Metastatic
Treatment for people who have received a prior anti-HER2-based regimen either:
in the metastatic setting.
in the neoadjuvant or setting and developed a recurrence during or within six months of completing treatment.
overexpression ()
Antibody-drug conjugate
Enhertu (fam-trastuzumab-deruxtecan-nxki)
Breast cancer
Metastatic
As treatment for people who have tumors that are HER2-low, received chemotherapy in the metastatic setting and whose cancer no longer responds to hormonal therapy.
HR-positive and HER2-low
Antibody-drug conjugate
Enhertu (fam-trastuzumab-deruxtecan-nxki)
Breast cancer
Metastatic
As treatment for people with HR-positive, HER2-low or HER2 ultralow that came back or got worse after one or more hormone therapies in the metastatic setting.
HR-positive and HER2-low or HER2-ultra low
Antibody-drug conjugate
Enhertu (fam-trastuzumab-deruxtecan-nxki)
Solid tumors (pan tumor)
Metastatic or unresectable
For adult patients with unresectable or metastatic, HER2-positive solid tumors (including colorectal cancer) who have received prior systemic treatment and have no alternative treatment options.
HER2 overexpression (HER2-positive)
Antibody-drug conjugate
Erbitux (cetuximab)
Colorectal cancer
Metastatic
Combined with FOLFIRI for first-line treatment, or combined with irinotecan for cancers that no longer respond to irinotecan-based chemotherapy or as a single agent in patients who have progressed after oxaliplatin- and irinotecan-based chemotherapy.
EGFR positive and KRAS mutation negative
EGFR inhibitor
Fruzaqla (fruquintinib)
Colorectal cancer
Metastatic
Used as a single agent when cancer has progressed after treatment with chemotherapy and targeted therapy.
No biomarker required
VEGF inhibitor
Herceptin (trastuzumab)
Breast cancer
Early stage
The treatment of Her2-positive breast cancer.
HER2 overexpression (HER2-positive)
Antibody targeting HER2 receptors
Herceptin (trastuzumab) and Tukysa (tucatinib) combination
Colorectal cancer
Metastatic or unresectable
For people who progressed after chemotherapy.
HER2 overexpression (HER2-positive)
Antibody targeting HER2 receptors and a kinase inhibitor that targets HER2 receptors
Ibrance (palbociclib)
Breast cancer
Metastatic
Combined with an aromatase inhibitor as treatment of advanced cancer as initial hormone therapy in postmenopausal women or in men.
HR-positive and HER2-negative
Targeted therapy known as a kinase inhibitor that blocks the CDK4/6 pathway
Ibrance (palbociclib)
Breast cancer
Metastatic
Combined with Faslodex (fulvestrant) as treatment in postmenopausal women or in men whose disease progressed following endocrine therapy.
HR-positive and HER2-negative
Targeted therapy known as a kinase inhibitor that blocks the CDK4/6 pathway
Kadcyla (trastuzumab emtansine)
Breast cancer
Early stage
Adjuvant therapy for people with early breast cancer who still have disease after neoadjuvant taxane and treatment with Herceptin
HER2 overexpression (HER2-positive)
Antibody targeting HER2 receptors
Kadcyla (trastuzumab emtansine)
Breast cancer
Metastatic
For treatment in people whose cancer got worse after receiving Herceptin and chemotherapy in the following settings:
for metastatic disease, or
as adjuvant therapy, and experienced disease recurrence during or within 6 months of completing adjuvant therapy.
HER2 overexpression (HER2-positive)
Antibody targeting HER2 receptors
Kisqali (ribociclib)
Breast cancer
Metastatic
Combined with an aromatase inhibitor for the treatment of pre/perimenopausal or postmenopausal women as initial hormone based therapy.
HR-positive and HER2-negative
Targeted therapy known as a kinase inhibitor that blocks the CDK4/6 pathway
Kisqali (ribociclib)
Breast cancer
Metastatic
Combined with Faslodex (fulvestrant) for the treatment of postmenopausal women, as initial hormone based therapy.
HR-positive and HER2-negative
Targeted therapy known as a kinase inhibitor that blocks the CDK4/6 pathway
Krazati (adagrasib)
Colorectal cancer
Metastatic
In combination with cetuximab for locally advanced or metastatic colorectal cancer (CRC) that has progressed after treatment with chemotherapy.
KRASG12C mutation
Targeted therapy against the KRASG12C protein
Lenvima (lenvatinib)
Endometrial cancer
Advanced disease
Combined with pembrolizumab, for the treatment of patients whose cancer has progressed after treatment and who are not candidates for surgery or radiation.
Tumors that are not MSI-H or (or ) - they may be referred to as MSI-Low, MSS, pMMR or MMR-P)
Targeted therapy known as a tyrosine kinase inhibitor
Mekinist (trametinib)
Melanoma
Unresectable or metastatic
As a single agent and in combination with dabrafenib for the treatment of unresectable or metastatic melanoma.
BRAF V600E or V600K tumor mutation
Targeted therapy known as a MEK inhibitor
Mekinist (trametinib)
Melanoma
Lymph node positive
Combined with Taflinar (dabrafenib) as adjuvant treatment of people with melanoma and involvement of lymph node(s), following complete resection.
BRAF V600E or V600K tumor mutation
Targeted therapy known as a MEK inhibitor
Mektovi (binimetinib)
Melanoma
Unresectable or metastatic
Combined with Braftovi (encorafenib), for the treatment of people with unresectable or metastatic melanoma.
BRAF V600E or V600K tumor mutation
Targeted therapy known as a MEK inhibitor
Orserdu (elacestrant)
Breast cancer
Metastatic
Used alone to treat men or postmenopausal women with HR-positive, HER2-negative breast cancer, which progressed after at least one line of hormone therapy therapy.
HR-positive, HER2-negative with an ESR1 mutation
Type of targeted hormonal therapy known as SERD (selective receptor degrader or downregulator)
Perjeta (pertuzumab)
Breast cancer
Locally advanced, inflammatory or early stage
Combined with Herceptin (trastuzumab) and docetaxel as treatment before surgery (neoadjuvant).
Before surgery (neoadjuvant) treatment for tumors larger than 2 cm or node-positive, or
After surgery (adjuvant) treatment for early breast cancer that has a high likelihood of coming back.
HER2 overexpression (HER2-positive)
Antibody targeting HER2 receptors
Piqray (alpelisib)
Breast cancer
Metastatic
Combined with Faslodex (fulvestrant) as treatment in men or post-menopausal women who progressed on or after treatment with hormone therapy.
HR-positive, HER2-negative and positive for a PIK3CA tumor mutation
Targeted therapy known as a kinase inhibitor that blocks the PIK3 pathway
Pluvicto (lutetium Lu 177 vipivotide tetraxetan)
Prostate cancer
Metastatic castration-resistant prostate cancer (mCRPC)
For treatment of mCRPC which has stopped responding or got worse after treatment with hormonal therapy using an androgen receptor inhibitor and taxane-based chemotherapy.
Imaging with a that looks for prostate cancers with the marker PSMA
Targeted radiation therapy (radioligand therapy)
Retevmo (selpercatinib)
Solid tumors (pan tumor)
Metastatic or unresectable
Second-line or later treatment for metastatic solid tumors for which there are no other treatment options.
RET fusion
Kinase inhibitor
Stivarga (regorafenib)
Colorectal cancer
Metastatic
For treatment of colorectal cancer that has progressed after treatment and for which there are no other treatment options.
No biomarker required
Targeted therapy known as a multi-kinase inhibitor
Tafinlar (dabrafenib)
Melanoma
Unresectable or metastatic
Combined with Mekinist (trametinib) for the treatment of people with unresectable or metastatic melanoma.
BRAF V600E or V600K tumor mutation
Targeted therapy known as a BRAF inhibitor
Tafinlar (dabrafenib)
Melanoma
Lymph node positive
Combined with Mekinist (trametinib) as adjuvant treatment of people with melanoma and involvement of lymph node(s), following complete resection.
BRAF V600E or V600K tumor mutation
Targeted therapy known as a BRAF inhibitor
Trodelvy (sacituzumab govitecan-hziy)
Breast cancer
Metastatic
For metastatic breast cancer that progressed, recurred or did not respond to at least two previous lines of treatment.
Triple-negative (, HER2-negative)
Antibody-drug conjugate (chemotherapy attached to antibody found in )
Truqap (capivasertib)
Breast cancer
Metastatic
Combined with fulvestrant as treatment for HR-positive, advanced or metastatic breast cancer which recurred or got worse after standard hormone therapy.
, PIK3 or AKT1 mutation in the tumor
Targeted therapy known as a kinase inhibitor that blocks the AKT pathway
Tukysa (tucatinib)
Breast cancer
Metastatic or unresectable
In combination with Herceptin (trastuzumab) to treat cancer which has progressed after at least one prior treatment with an anti-HER2 treatment in the metastatic setting.
HER2 overexpression (HER2-positive)
Targeted therapy known as a kinase inhibitor that targets HER2 receptors
Vectibix (panitumumab)
Colorectal cancer
Metastatic
Combined with FOLFOX for first-line treatment.
Negative for KRAS and NRAS mutations
Targeted therapy that targets a receptor known as EGFR
Vectibix (panitumumab)
Colorectal cancer
Metastatic
As a single therapy following disease progression after prior treatment with fluoropyrimidine, oxaliplatin, and irinotecan-containing chemotherapy.
Negative for KRAS and NRAS mutations
Targeted therapy that targets a receptor known as EGFR
Verzenio (abemaciclib)
Breast cancer
Metastatic
Used alone to treat breast cancer that has progressed after treatment with hormone therapy and chemotherapy in the metastatic setting.
HR-positive and HER2-negative
Targeted therapy known as a kinase inhibitor that blocks the CDK4/6 pathway
Verzenio (abemaciclib)
Breast cancer
Metastatic
Combined with Faslodex (fulvestrant) as treatment in women whose disease progressed following endocrine therapy.
HR-positive and HER2-negative
Targeted therapy known as a kinase inhibitor that blocks the CDK4/6 pathway
Vitrakvi (larotrectinib)
Solid tumors (pan tumor)
Metastatic or unresectable
For treatment in metastatic solid tumors for which there are no other treatment options.
NTRK fusion
Kinase inhibitor
Xofigo (Radium 223 dichloride)
Prostate cancer
Metastatic castration-resistant prostate cancer (mCRPC)
For treatment of mCRPC that has spread to the bones but has not to other organs.
No biomarker needed
Targeted radiation therapy (radioligand therapy)
Zelboraf (vemurafenib)
Melanoma
Unresectable or metastatic
Combined with Tecentriq (atezolizumab) and Cotellic (cobimetinib) in people with melanoma that has the BRAF gene mutation, when the cancer can’t be removed by surgery or has spread to other parts of the body.
