The US Food and Drug Administration (FDA) granted accelerated approval to rucaparib (Rubraca), a PARP inhibitor, for the treatment of women with deleterious BRCA mutation-associated ovarian cancer. The approval is for women who have been treated with at least two prior chemotherapy regimens.

“Recurrent ovarian cancer remains one of the most difficult cancers to treat and for so many years, medical advances in this space have been limited,” said Robert L. Coleman, MD, of the MD Anderson Cancer Center in Houston, in a press release from the drug’s developer. Coleman was one of the principal investigators in the trials leading to rucaparib’s approval.

The FDA also approved a diagnostic in conjunction with the new agent, the FoundationFocus CDxBRCA test. This is the first next-generation sequencing test approved by the FDA to test for mutations in ovarian cancer, and can detect both BRCA1 and BRCA2 alterations.

The drug’s approval, after being granted Priority Review status, was based on two multicenter, single-arm, open-label trials, in a total of 106 patients with advanced ovarian cancer whose disease progressed after treatment with at least two prior therapies. The newly approved diagnostic was able to retrospectively verify BRCA mutation status previously determined in 96% of patients for whom a tissue sample was available.

The objective response rate (ORR) with rucaparib was 54%, and the median duration of response in the 57 patients with a response was 9.2 months. Patients deemed platinum-sensitive had an ORR of 66%, compared with only 25% in platinum-resistant patients, and 0% in platinum-refractory patients. There were no differences in response rate between those with BRCA1 and BRCA2 mutations.

A safety cohort of 377 patients showed the most common adverse events included nausea, fatigue, vomiting, anemia, and others. Adverse events led to dose discontinuation in 10% of patients, most frequently from fatigue/asthenia. Two of the 377 patients (0.5%) developed a myelodysplastic syndrome, and two patients in another rucaparib trial developed acute myeloid leukemia. The FDA approval noted that patients should be monitored for hematologic toxicity at baseline and monthly after treatment begins.

“Today’s approval of Rubraca for the treatment of advanced ovarian cancer demonstrates the value of treatment with PARP inhibitors and represents an important advance for women diagnosed with either germline or somatic BRCA-mutated tumors who have been treated with two or more chemotherapies,” Coleman said.