December 19, 2016

FDA Approves Rucaparib to Treat Advanced Ovarian Cancer

ROCKVILLE, Md -- December 19, 2016 -- The US Food and Drug Administration (FDA) has approved rucaparib (Rubraca) to treat women with advanced ovarian cancer who have been treated with 2 or more chemotherapies and whose tumours have a specific gene mutation (deleterious BRCA) as identified by an FDA-approved companion diagnostic test.

“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, MD, FDA’s Center for Drug Evaluation and Research, Rockville, Maryland. “Women with these gene abnormalities who have tried at least 2 chemotherapy treatments for their ovarian cancer now have an additional treatment option.”

Rucaparib is a poly ADP-ribose polymerase (PARP) inhibitor that blocks an enzyme involved in repairing damaged DNA. By blocking this enzyme, the DNA inside the cancerous cells with damaged BRCA genes may be less likely to be repaired, leading to cell death and possibly a slow-down or stoppage of tumour growth.

The FDA also approved the FoundationFocus CDxBRCA companion diagnostic for use with rucaparib, which is the first next-generation-sequencing (NGS)-based companion diagnostic approved by the agency. The NGS test detects the presence of deleterious BRCA gene mutations in the tumour tissue of ovarian cancer patients. If one or more of the mutations are detected, the patient may be eligible for treatment with rucaparib.

The safety and efficacy of rucaparib were studied in 2, single-arm clinical trials involving 106 participants with BRCA-mutated advanced ovarian cancer who had been treated with 2 or more chemotherapy regimens. BRCA gene mutations were confirmed in 96% of tested trial participants with available tumour tissue using the FoundationFocus CDxBRCA companion diagnostic. The trials measured the percentage of participants who experienced complete or partial shrinkage of their tumours (overall response rate). Of the patients who received rucaparib, 54% experienced complete or partial shrinkage of their tumours lasting a median of 9.2 months.

Common side effects of rucaparib include nausea, fatigue, vomiting, anaemia, abdominal pain, dysgeusia, constipation, decreased appetite, diarrhoea, thrombocytopenia, and dyspnoea. Rucaparib is associated with serious risks, such as myelodysplastic syndrome, acute myeloid leukaemia, and fetal harm.

The FDA approved rucaparib 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 endpoint that is reasonably likely to predict clinical benefit. The sponsor is continuing to study this drug in patients with advanced ovarian cancer who have BRCA gene mutations and in patients with other types of ovarian cancer. The FDA also granted the rucaparib application breakthrough therapy designation and priority review status. Rucaparib also received orphan drug designation, which provides incentives such as tax credits, user fee waivers and eligibility for exclusivity to assist and encourage the development of drugs intended to treat rare diseases.

SOURCE: US Food and Drug Administration

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