On March 27, 2017, the FDA accelerated the approval of niraparib (Zejula; Tesaro), a PARP inhibitor, for maintenance treatment of 3 types of cancers—recurrent epithelial ovarian cancer, fallopian tube cancer, or primary peritoneal cancer—in adults who had a complete or partial 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 FDA’s Center for Drug Evaluation and Research and Director of the FDA’s Oncology Center of Excellence. “Zejula 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,” he added.
The FDA approval was based on a randomized clinical trial of 553 patients with recurrent epithelial ovarian cancer, fallopian tube cancer, or primary peritoneal cancer, who had received ≥2 platinum-based chemotherapies and had a complete or a partial response to the most recent chemotherapy regimen.
All patients were tested for BRCA mutation with the FDA-approved BRACAnalysis CDx test. The median progression-free survival (PFS) was 21 months in patients with a BRCA mutation who received niraparib versus 5.5 months in patients who received placebo. Among patients without a BRCA mutation, the median PFS was 9.3 months with niraparib versus 3.9 months with placebo.
The most common (≥10%) all-grade adverse events with niraparib were thrombocytopenia, anemia, neutropenia, leukopenia, palpitations, nausea, constipation, vomiting, abdominal pain or distention, mucositis or stomatitis, diarrhea, dyspepsia, dry mouth, fatigue/asthenia, decreased appetite, urinary tract infection, elevations in AST or ALT levels, myalgia, back pain, arthralgia, headache, dizziness, dysgeusia, insomnia, anxiety, nasopharyngitis, dyspnea, cough, rash, and hypertension.
The most common grade 3 or 4 adverse reactions included neutropenia, nausea, constipation, diarrhea, urinary tract infection, elevations in AST or ALT levels, dyspnea, and hypertension.
Niraparib received a breakthrough therapy designation for these 3 types of cancer, and an orphan drug designation for recurrent epithelial ovarian cancer.