Ovarian Cancer Treatment Receives Accelerated Approval from Food and Drug Administration
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Ovarian Cancer Treatment Receives Accelerated Approval from Food and Drug Administration

Treatment is for women who have advanced cancer with a particular gene mutation

December 19, 2016

The Food and Drug Administration (FDA) has granted accelerated approval to Rubraca, a drug meant for treating women who have a certain kind of ovarian cancer.

The drug has been approved for women who have advanced ovarian cancer, have already undergone treatment with two or more chemotherapies, and whose tumors have a particular gene mutation (deleterious BRCA) identified by a companion diagnostic test approved by the FDA.

"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," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research and acting director of the FDA's Oncology Center of Excellence. "Women with these gene abnormalities who have tried at least two chemotherapy treatments for their ovarian cancer now have an additional treatment option."

According to estimates made by the National Cancer Institute, 22,280 women will be diagnosed with ovarian cancer in 2016. An estimated 14,240 of these will die of the disease.

Roughly 15 to 20 percent of patients who have ovarian cancer have a BRCA gene mutation. Normally, this type of gene helps repair damaged DNA and work to prevent tumors from developing. Mutations of the gene, however, can result in certain cancers, including ovarian cancers.

Rubraca blocks a certain enzyme that helps repair damaged DNA. When this enzyme is blocked, DNA in the cancerous cells with mutated genes may be less likely to be repaired, resulting in the death of the cells and possibly a slowing or stoppage of tumor growth.

The safety and effectiveness of the drug were studied in two single-arm clinical studies—studies in which all participants received the same treatment—that involved 106 patients. Each of these patients had BRCA-mutated advanced ovarian cancer and had been treated with two or more chemotherapy regimens. The mutations were confirmed in 96 percent of trial participants tested using available tumor tissue.

The trials measured how many participants experienced either complete or partial shrinkage of their tumors. Fifty-four percent who received Rubraca in the trials experienced such shrinkage, lasting for a median of 9.2 months.

The common side effects of Rubraca include nausea, fatigue, vomiting, low levels of red blood cells, abdominal pain, unusual taste sensation, constipation, decreased appetite, diarrhea, low levels of blood platelets, and trouble breathing. Rubraca is associated with serious risks, such as problems with bone marrow, a kind of blood cancer called acute myeloid leukemia, and fetal harm.