Accelerated Approval of Rucaparib to Treat Ovarian Cancer

January 3, 2017

We all know the devastating effects of ovarian cancer on women globally. With the amount of women being diagnosed with or dying from the disease each year, it is clear that more drugs need to be approved for its treatment.

We all know the devastating effects of ovarian cancer on women globally. With the amount of women being diagnosed with or dying from the disease each year, it is clear that more drugs need to be approved for its treatment.

On December 19, 2016, the US Food and Drug Administration (FDA) granted accelerated approval to rucaparib (Rubraca), a PARP inhibitor, for the treatment of advanced ovarian cancer and tumors which have a deleterious BRCA gene mutation.1 To be able to receive this drug, women must also have received two alternate forms of chemotherapy prior to treatment with rucaparib and have undergone tumor gene testing with the use of an FDA-approved companion diagnostic test, such as the approved FoundationFocus CDxRCA diagnostic test, which also received FDA approval on December 19, 2016.

This accelerated approval will give hope to women who would otherwise have limited options. Two, single-arm clinical trials (n = 106) studying rucaparib reported a 54% overall response rate for an average of 9.2 months, measured by complete or partial tumor response (shrinkage) using the RECIST version 1.1 criteria, with 9% achieving complete response and 45% achieving partial response; nine patients experienced disease progression.2, 3

One of the two trials only enrolled women who were deemed platinum-sensitive, whereas the other trial (ARIEL2) accepted subjects who were deemed platinum-sensitive or had relapsed.

Side effects experienced during treatment included those common to other treatments: nausea, fatigue, vomiting, anemia, abdominal pain, altered taste, constipation, loss of appetite, diarrhea, thrombocytopenia, elevated ALT/AST, and dyspnea. Some women did experience more serious side effects such as myelodysplastic syndrome and harm to a growing fetus.

Additional research is being conducted to evaluate the use of rucaparib in other advanced ovarian cancers and those with BRCA gene mutations.

References:

1. US Food and Drug Administration. FDA grants accelerated approval to new treatment for advanced ovarian cancer. 2016 Dec 19.
2. ClinicalTrials.gov. A Study of Rucaparib in Patients With Platinum-Sensitive, Relapsed, High-Grade Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer (ARIEL2) (ARIEL2).
3. ClinicalTrials.gov. A Study of Oral Rucaparib in Patients With a Solid Tumor (Phase I) or With gBRCA Mutation Ovarian Cancer (Phase II).