PARP inhibitor · small molecule

rucaparib

0 active trials, targeting PARP. 2 active approvals for a biomarker on marked.bio.

Also known as CO-338, AG-014699, Rubraca.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

Therapy	Biomarker	Tumour	United States	European Union
rucaparib FDA, 2016 deleterious BRCA, after >=2 chemotherapies AA 19/12/2016; converted to regular approval 06/04/2018 (FDA Verified clinical benefit list). No longer on the current label (12/2022), so it is withdrawn; the exact withdrawal date was not checked against an FDA source. Source FDA, 2018 maintenance, deleterious BRCA-mutated (germline and/or somatic) recurrent ovarian cancer in response to platinum Approved 2018 for all patients; later limited to BRCA-mutated (agent recorded 2022). Year = first approval in this setting. Label Recent Major Changes show Indications 1.1 changed 12/2022, restricting recurrent-disease maintenance (previously all patients) to BRCA-mutated. Date is from the label, not an FDA announcement. Source EU (EMA), 2018 treatment of platinum-sensitive relapsed or progressive BRCA-mutated high-grade ovarian cancer after >=2 platinum lines, unable to tolerate further platinum Part of the initial conditional MA (23/05/2018). Removed after an Article 20 review of ARIEL4, which showed a detrimental effect on overall survival (CHMP opinion 21/07/2022, EC decision 21/09/2022). FDA equivalent: A127. Source Rubraca	BRCA1/2 mutation	ovarian	Approved 2018	Withdrawn approved 2018
ARIEL3 (BRCA-mutated cohort) vs placebo, 130 vs 66 patients PFS 16.6 months vs 5.4 months, HR 0.23 (0.16-0.34) Stopped for side effects 15% Trial details and side effects Patients maintenance, BRCA-mutated recurrent ovarian cancer after response to platinum (NCT01968213) Most common side effects nausea 79%, fatigue/asthenia 74%, abdominal pain 48%, rash 45%, anaemia 41%, constipation 39% (129 patients) Notes Rubraca US label rev. 12/2025. Source US label
rucaparib FDA, 2020 mCRPC, deleterious BRCA mutation (germline and/or somatic), previously treated with androgen receptor-directed therapy AA 15/05/2020 (after AR-directed therapy and a taxane); converted to regular approval 17/12/2025 based on TRITON3, and the taxane requirement was dropped. DailyMed label (12/2022) not yet updated. Source Rubraca	BRCA1/2 mutation	prostate	Approved 2020	Not approved
TRITON2 single-arm, 62 with measurable disease (115 enrolled) Response 44% (31-57)Lasting not reached Stopped for side effects 8%Fatal 1.7% Trial details and side effects Patients BRCA-mutated mCRPC after androgen receptor-directed therapy and a taxane (NCT02952534) Most common side effects fatigue 62%, nausea 52%, anaemia 43%, decreased appetite 28%, rash 27%, constipation 27% (115 patients) Later trial TRITON3 BRCA subgroup vs androgen receptor-directed therapy or docetaxel (201 vs 101 patients): rPFS 11.2 vs 6.4 months, HR 0.50 (0.36-0.69). In the US label. Notes Rubraca US label rev. 12/2025. Source US label

Biomarkers

Biomarker	Active trials	All trials	Furthest active phase
BRCA1/2 mutation	0	15	–

Trials

0 of 15 trials shown

ARIEL4: A Study of Rucaparib Versus Chemotherapy BRCA Mutant Ovarian, Fallopian Tube, or Primary Peritoneal Cancer Patients

Completed Phase 3 pharmaand Started 2017 NCT02855944

cisplatincarboplatinpaclitaxelgemcitabinerucaparib FDA

Note: biopsy is optional for patients known to harbor a BRCA1/2 mutation

BRCA1/2 mutationOvarian Cancer, Epithelial Ovarian Cancer, Fallopian Tube Cancer, Peritoneal Cancer
Rucaparib in Treating Patients With Genomic LOH High and/or Deleterious BRCA1/2 Mutation Stage IV or Recurrent Non-small Cell Lung Cancer (A Lung-MAP Treatment Trial)

Completed Phase 2 SWOG Cancer Research Network Started 2019 NCT03845296

rucaparib FDA

S1900A biomarker eligibility defined as LOH high and/or deleterious BRCA1/2 mutation is as follows using the Foundation Medicine Inc (FMI) tissue- assay:

BRCA1/2 mutationDeleterious BRCA1 Gene Mutation, Deleterious BRCA2 Gene Mutation, Loss of Heterozygosity, Lung Non-Small Cell Squamous Carcinoma
A Study to Evaluate Rucaparib in Combination With Other Anticancer Agents in Participants With a Solid Tumor (SEASTAR)

Terminated Phase 1/2 pharmaand Started 2019 NCT03992131

rucaparib FDALucitanibsacituzumab govitecan

…cancer OR urothelial carcinoma OR ovarian cancer OR have a solid tumor with a deleterious mutation in BRCA1, BRCA2, PALB2, RAD51C or RAD51D

BRCA1/2 mutationOvarian Cancer, Triple-negative Breast Cancer, Urothelial Carcinoma, Solid Tumor
Liposomal Irinotecan, Fluorouracil, Leucovorin Calcium, and Rucaparib in Treating Patients With Metastatic Pancreatic, Colorectal, Gastroesophageal, or Biliary Cancer

Unknown status Phase 1/2 Academic and Community Cancer Research United Started 2018 NCT03337087

fluorouracilirinotecanliposomal irinotecanleucovorinrucaparib FDA

…pancreas with genomic markers (signature) of homologous recombination deficiency (HRD) or BRCA1 or BRCA2 or PALB2 mutation, or HRD (non-BRCA, non-PALB) who have not received any systemic therapy in the metastatic…

BRCA1/2 mutationMetastatic Biliary Tract Carcinoma, Metastatic Colorectal Carcinoma, Metastatic Gastroesophageal Junction Adenocarcinoma, Metastatic Malignant Digestive System Neoplasm
Docetaxel, Carboplatin, and Rucaparib Camsylate in Treating Patients With Metastatic Castration Resistant Prostate Cancer With Homologous Recombination DNA Repair Deficiency

Terminated Phase 2 University of Washington Started 2018 NCT03442556

carboplatindocetaxelrucaparib FDA

Presence of pathogenic or likely pathogenic germline mutation/variant in BRCA2, BRCA1, ATM or PALB2

BRCA1/2 mutationATM Gene Mutation, BRCA1 Gene Mutation, BRCA2 Gene Mutation, Castration Levels of Testosterone
A Study of Rucaparib in Patients With Metastatic Castration-resistant Prostate Cancer and Homologous Recombination Gene Deficiency

Completed Phase 2 pharmaand Started 2017 NCT02952534

rucaparib FDA

Have a deleterious mutation in BRCA1/2 or ATM, or molecular evidence of other homologous recombination deficiency

BRCA1/2 mutationMetastatic Castration Resistant Prostate Cancer
Maintenance Rucaparib in BRCA1, BRCA2 or PALB2 Mutated Pancreatic Cancer That Has Not Progressed on Platinum-based Therapy

Terminated Phase 2 Abramson Cancer Center at Penn Medicine Started 2017 NCT03140670

rucaparib FDA

Documented deleterious BRCA1/2 or PALB2 mutation (germline or somatic) as assessed by CLIA certified laboratory.

BRCA1/2 mutationPancreatic Cancer
A Study of Rucaparib in Patients With Pancreatic Cancer and a Known Deleterious BRCA Mutation

Completed Phase 2 pharmaand Started 2014 NCT02042378

rucaparib FDA

Documented deleterious or suspected deleterious (or equivalent interpretation) BRCA mutation (germline or somatic) as assessed by a local laboratory

BRCA1/2 mutationPancreatic Cancer, Pancreatic Ductal Adenocarcinoma
A Study of Rucaparib in Patients With Platinum-Sensitive, Relapsed, High-Grade Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer (ARIEL2)

Completed Phase 2 pharmaand Started 2013 NCT01891344

rucaparib FDA

Note: biopsy is optional for patients known to harbor a deleterious gBRCA mutation

BRCA1/2 mutationOvarian Cancer, Epithelial Ovarian Cancer, Fallopian Tube Cancer, Peritoneal Cancer
A Study of Oral Rucaparib in Patients With a Solid Tumor (Phase I) or With gBRCA Mutation Ovarian Cancer (Phase II)

Completed Phase 1/2 pharmaand Started 2011 NCT01482715

rucaparib FDA

Have a known deleterious BRCA mutation (gBRCA or sBRCA) (as determined by a local laboratory that has received an international …

BRCA1/2 mutationOvarian Cancer, Fallopian Tube Cancer, Peritoneal Cancer, Advanced Solid Tumor With Evidence of Germline or Somatic BRCA
PARP Inhibition for Triple Negative Breast Cancer (ER-/PR-/HER2-)With BRCA1/2 Mutations

Completed Phase 2 Hoosier Cancer Research Network Started 2010 NCT01074970

cisplatinrucaparib FDA

PARP Inhibition for Triple Negative Breast Cancer (ER-/PR-/HER2-)With BRCA1/2 Mutations

BRCA1/2 mutationBreast Cancer
Rucaparib(CO-338;Formally Called AG-014699 or PF-0136738) in Treating Patients With Locally Advanced or Metastatic Breast Cancer or Advanced Ovarian Cancer

Completed Phase 2 Cancer Research UK Started 2007 NCT00664781

rucaparib FDAprotein expression analysiswestern blotting

…ers of a mutation of BRCA1 or BRCA2 or considered to be highly likely to be carriers of a BRCA1 or 2 mutation\* (score of ≥ 20 as per Manchester criteria) and have histologically documented locally a…

BRCA1/2 mutationbrca1 Mutation Carrier, brca2 Mutation Carrier, Breast Cancer, Ovarian Cancer
A Study of Rucaparib in Japanese Patients With a Previously-treated Solid Tumor

Completed Phase 1 pharmaand Started 2018 NCT03499444

rucaparib FDA

…patients enrolled in the dose-expansion portion, has high-grade serous ovarian cancer, or BRCA 1/2 mutated breast cancer, or other solid tumor with BRCA 1/2 or related gene mutation

BRCA1/2 mutationAdvanced Solid Tumor
A Combination Study of Rucaparib and Atezolizumab in Participants With Advanced Gynecologic Cancers and Triple-Negative Breast Cancer

Completed Phase 1 Roche Started 2017 NCT03101280

atezolizumabrucaparib FDA

…LY, have a deleterious germline or somatic breast cancer susceptibility gene 1 (BRCA1) or BRCA2 mutation or tumors that are wild-type BRCA but show high levels of loss of heterozygosity (LOH) (t…

BRCA1/2 mutationGynecologic Neoplasms
Absorption, Metabolism, and Excretion Following a Single Oral Dose of [14C]-Rucaparib

Completed Phase 1 pharmaand Started 2016 NCT02986100

rucaparib FDA

Part II only: Have a known deleterious BRCA1/2 mutation (germline or somatic) as determined by a local or central laboratory

BRCA1/2 mutationSolid Tumor