The U.S. Food and Drug Administration (FDA) granted accelerated approval to Rubraca (rucaparib) in December 2016 for women with advanced ovarian cancer. Patients who have undergone two or more chemotherapies and whose tumors have the BRCA gene mutation are approved for Rubraca.
According to the National Cancer Institute, of the more than 22,000 women diagnosed each year with ovarian cancer, 15 to 20 percent have the BRCA gene mutation. In normal women, BRCA genes repair damaged DNA and actually work to prevent tumor development. Mutated genes, however, can allow tumors to develop.
Rubraca functions as an inhibitor, blocking an enzyme that repairs damaged DNA in cancerous cells. When their DNA is not repaired, the cancerous cells may die and tumor growth may be slowed down or stopped.
The FDA also approved the diagnostic FoundationFocus CDxBRCA, the first next-generation-sequencing-based companion diagnostic tool ever to gain FDA approval, to be used with Rubraca. This tool will be used to see if an ovarian cancer patient has the BRCA gene mutation and qualifies for Rubraca treatment.
In clinical trials, 54 percent of the participants who received Rubraca experienced complete or partial shrinkage of their tumors, lasting just over nine months. Like other chemotherapy treatments, Rubraca has side effects, including nausea, fatigue, abdominal pain, and breathing problems. Serious risks include bone marrow problems and cancer of the blood.
Rubraca is marketed by Clovis Oncology, Inc., a company based in Boulder, Colorado. The FoundationFocus companion diagnostic is marketed by Foundation Medicine, Inc., a company based in Cambridge, Massachusetts.
The FDA not only approved Rubraca under its accelerated approval program, but it also granted the Rubraca application breakthrough therapy designation, priority review status, and orphan drug designation. Orphan drug designation means the drug treats a rare disease or condition. This designation provides incentives such as tax credits and fee waivers meant to encourage the development of drugs intended to treat rare diseases.