The U.S. Food and Drug Administration granted Rubraca (rucaparib) accelerated approval as a treatment option for an advanced ovarian cancer. Patients who can use Rubraca are to be identified by a companion diagnostic test that the agency also approved.
Specifically, women who may be prescribed Rubraca are patients who have undergone at least two chemotherapies and who have tumors with deleterious BRCA, a specific mutation.
"Today's approval is another example of the trend we are seeing in developing targeted agents to treat cancers caused by specific mutations in a patient's genes," Richard Pazdur, M.D., the FDA's Office of Hematology and Oncology director, said.
According to estimates by the National Cancer Institute, some 22,280 women will receive ovarian cancer diagnoses in 2016, while 14,240 will die from the disease. Out of all ovarian cancer patients, between 15 and 20 percent have the deleterious BRCA mutation.
Rubraca And BRCA Gene Function
Generally, BRCA genes are actually involved in the repair work needed by damaged DNA as well as the prevention of tumor development. However, mutations in the gene cause certain types of cancer.
As a poly ADP-ribose polymerase inhibitor, Rubraca works by blocking an enzyme tasked with repairing DNA damage. With this enzyme blocked, DNA within cancer cells are unlikely to undergo repair, resulting in cell death that can either slow down tumor growth or stop it completely.
Rubraca Companion Diagnostic Test
Alongside Rubraca's approval, the FoundationFocus CDxBRCA was also approved by the FDA as a companion diagnostic test based on next-generation sequencing (NGS). As an NGS test, it detects deleterious BRCA in the tumor tissues of patients with ovarian cancer. When at least one mutation is detected in a patient, they become eligible to receive treatment using Rubraca.
Rubraca Side Effects
Two clinical trials were carried out to test for the efficacy and safety of Rubraca. Based on results, 54 percent of participants experienced tumor shrinkage, but some side effects were also observed, including trouble breathing, low blood platelet count, diarrhea, decreased appetite, constipation, unusual taste sensation, abdominal pains, low red blood cell count, vomiting, fatigue, and nausea. Serious risks associated with the drug, on the other hand, include fetal harm, acute myeloid leukemia (a type of blood cancer), and bone marrow problems.
Rubraca and the companion diagnostic test FoundationFocus CDxBRCA are marketed by Colorado-based Clovis Oncology, Inc. and Massachusetts' Foundation Medicine, Inc., respectively.
Aside from accelerated approval, priority review status and breakthrough therapy and orphan drug designations were granted for Rubraca by the FDA.