Dive Brief:

• FDA on Monday approved AstraZeneca and Merck & Co.'s Lynparza for use in treating pancreatic cancer. The oral pill can now be prescribed for a small share of patients with a specific mutation that also increases the risk of breast and ovarian cancer.
• The agency simultaneously awarded a premarket approval supplement to Myriad Genetics' BRACAnalysis test as a companion diagnostic for the new indication. The test first gained FDA approval in 2014 to identify ​patients with advanced ovarian cancer eligible to receive Lynparza as a fourth-line treatment.
• The OK for the new Lynparza indication was based on a study that found the drug, when used after chemotherapy, kept patients from dying or having their disease progress for almost twice as long as patients taking a placebo. No overall survival benefit was initially observed, however, tempering the significance of the trial's success. National treatment guidelines were revised in July 2019 to recommend use of Lynparza in this patient group. An FDA advisory committee voted 7-5 in favor of approval earlier in December.

Dive Insight:

While other types of cancer have seen improvements in treatment thanks to use of targeted therapies or immuno-oncology agents, pancreatic cancer has proven more challenging. Older chemotherapy and radiotherapy combinations are still the main form of treatment at most stages of the disease.

Lynparza (olaparib) is the second targeted drug approved in pancreatic cancer after Roche's Tarceva (erlotinib), which won an OK in 2005. Lynparza can be used in 4% to 7% of pancreatic cancer patients with mutations in a gene known as BRCA — similar to the proportion of patients for whom the pill can be prescribed in ovarian and breast cancer.

Earlier this year, AstraZeneca detailed the results of the POLO trial, which enrolled 154 BRCA-positive patients with metastatic disease who didn't see their tumors grow or spread after 16 weeks of platinum-based chemotherapy.

The study's aim was to test whether giving patients Lynparza could further stave off subsequent disease progression, an approach to treatment known as maintenance therapy.

Patients taking Lynparza were 47% less likely to die or see their disease progress than those taking placebo, results showed. That translated to a median of 7.4 months of so-called progression-free survival on Lynparza, compared to 3.8 months for patients who received placebo.

Trial investigators conducted an early analysis of overall survival, but found no significant difference between patients on Lynparza versus those on placebo. In both groups, median overall survival stretched past 18 months from when patients entered the study and were randomized to treatment.

When this analysis was prepared, 41 of 90 patients taking Lynparza and 30 of 61 patients taking a placebo had died. As analysis of these patients continues, it is possible that investigators will be able to detect a difference in overall survival between the two groups.

Following the presentation of POLO data at the American Society for Clinical Oncology meeting, the National Comprehensive Cancer Network in July added Lynparza to its guidelines for pancreatic cancer. Trial investigators also published results in the New England Journal of Medicine in July.

Lynparza is one of three fast-growing oncology drugs helping to drive revenue growth for AstraZeneca. Sales over the first nine months of the year nearly doubled compared to the same period in 2018, reaching $847 million through September.

Two similar drugs to Lynparza, GlaxoSmithKline's Zejula (niraparib) and Clovis Oncology's Rubraca (rucaparib), are also being studied in pancreatic cancer.

Since 2014, Myriad's BRACAnalysis test has also been approved by FDA as a complementary diagnostic both for identifying patients with germline BRCAm metastatic breast cancer previously treated with chemotherapy and eligible for treatment with Lynparza, as well as for patients with recurrent platinum-sensitive germline BRCAm ovarian cancer eligible for maintenance treatment with the drug.

FDA also approved BRACAnalysis as a companion diagnostic to identify patients newly diagnosed with advanced ovarian cancer who are eligible for first-line maintenance treatment with the drug.