RDR: FDA Approves Rubraca (rucaparib) for Rare Ovarian Cancer

December 28, 2016

The FDA has approved the orphan drug Rubraca (rucaparib) to treat women with advanced ovarian cancer who have been treated with 2 or more chemotherapies and whose tumors have a specific gene mutation (deleterious BRCA) as identified by an FDA-approved companion diagnostic test.

Approximately 15-20% of patients with ovarian cancer have a BRCA gene mutation. The National Cancer Institute estimates that 22,280 women will be diagnosed with ovarian cancer in 2016 and an estimated 14,240 will die of this disease.

To aid in diagnosing patients with the BRCA gene mutation, the FDA also approved the FoundationFocus CDxBRCA companion diagnostic for use with Rubraca. The test detects the presence of deleterious BRCA gene mutations in the tumor tissue of ovarian cancer patients. If one or more of the mutations are detected, the patient may be eligible for treatment with.

Accelerated Approval

The drug was given accelerated approval based on data from 106 patients across 2 trials. In a pooled analysis of data from the studies, the objective response rate (ORR) was 54% (95% CI, 44-64) with Rubraca lasting a median of 9.2 months.

Rubraca was approved under its accelerated approval program, which allows approval of a drug to treat a serious or life-threatening disease or condition based on clinical data showing the drug has an effect on a surrogate (substitute) endpoint that is reasonably likely to predict clinical benefit. The drug also had breakthrough therapy designation, priority review status, and orphan drug designation.


Common side effects of Rubraca include nausea, fatigue, vomiting, low levels of red blood cells (anemia), abdominal pain, unusual taste sensation (dysgeusia), constipation, decreased appetite, diarrhea, low levels of blood platelets (thrombocytopenia) and trouble breathing (dyspnea). Rubraca is associated with serious risks, such as bone marrow problems (myelodysplastic syndrome), a type of cancer of the blood called acute myeloid leukemia and fetal harm.

Rubraca is marketed by Clovis Oncology, Inc. The FoundationFocus CDxBRCA companion diagnostic is marketed by Foundation Medicine, Inc. .

Thanks to Rare Disease Report for this story.

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