The FDA approved niraparib for the maintenance treatment (intended to delay cancer growth) of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer whose tumors have completely or partially shrunk (complete or partial response, respectively) in response to platinum-based chemotherapy.
"Maintenance therapy is an important part of a cancer treatment regimen for patients who have responded positively to a primary treatment," said Richard Pazdur, MD, acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research and director of the FDA's Oncology Center of Excellence. "[Niraparib] offers patients a new treatment option that may help delay the future growth of these cancers, regardless of whether they have a specific genetic mutation."
Niraparib is a poly ADP-ribose polymerase (PARP) inhibitor that blocks an enzyme involved in repairing damaged DNA. By blocking this enzyme, DNA inside the cancerous cells may be less likely to be repaired, leading to cell death and possibly a slow-down or stoppage of tumor growth.
The safety and efficacy of niraparib were studied in a randomized trial of 553 patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who had received at least two prior treatments of platinum-based chemotherapy and who had experienced a complete or partial response to their most recent chemotherapy treatment. Patients were tested with an FDA-approved test to determine whether they had a specific gene mutation, called a deleterious or germline BRCA mutation. The trial measured the length of time the patients' tumors did not grow after treatment (progression-free survival) in patients with and without the mutation. The median progression-free survival for patients taking niraparib who had a germline BRCA mutation was 21 months compared to 5.5 months for the same patient population taking a placebo. The median progression-free survival for patients taking niraparib who did not have a germline BRCA mutation was 9.3 months compared to 3.9 months for the same patient population taking a placebo.
Common side effects of niraparib include anemia, thrombocytopenia, neutropenia or leukopenia, heart palpitations, nausea, constipation, vomiting, distention, mucositis, diarrhea, dyspepsia, dry mouth, fatigue, decreased appetite, urinary tract infection, AST/ALT elevation, myalgia, back pain, arthralgia, headache, dizziness, dysgeusia, insomnia, anxiety, nasopharyngitis, dyspnea, cough, rash, and high blood pressure (hypertension). Niraparib is associated with serious risks, such as hypertension, hypertensive crisis, myelodysplastic syndrome, acute myeloid leukemia, and bone marrow suppression.Women who are pregnant or breastfeeding should not take niraparib because it may cause harm to a developing fetus or a newborn baby.
The FDA granted this application Fast Track, Priority Review and Breakthrough Therapy designations.
Niraparib also received Orphan Drug designation specifically for its use in treating recurrent epithelial ovarian cancer. This designation provides incentives to assist and encourage the development of drugs for rare diseases.