December 23, 2014 — FDA on Friday approved AstraZeneca's Lynparza, a first-of-its-kind treatment for ovarian cancer associated with mutations of the BRCA1 and BRCA2 genes, the Wall Street Journal reports.
According to the Journal, BRCA1 and BRCA2 mutations are associated with up to 15% of ovarian cancer cases and up to 10% of breast cancer cases. Genetic tests are available to assess cancer risk associated with the gene mutations, but no treatment specifically for cancers associated with the mutations has been available.
Details of FDA Approval
FDA granted accelerated approval to Lynparza, which has the generic name of olaparib, after a mid-stage study found that it shrunk tumors in 34% of patients. FDA said the drug's continued approval will be contingent on late-stage studies verifying its safety and effectiveness (Walker, Wall Street Journal, 12/19).
The agency approved the drug along with a "companion diagnostic," Myriad Genetics' BRACAnalysis CDx test. Patients must undergo the test to demonstrate that they have a specific BRCA gene mutation prior to receiving Lynparza (Healy, "Science Now," Los Angeles Times, 12/19). In addition, FDA approved the drug only for patients who have tumors that are no longer responding to chemotherapy (Wall Street Journal, 12/19).
New Type of Treatment
Lynparza is the first treatment approved in a class of drugs known as poly ADP-ribose polymerase, or PARP, inhibitors. Such drugs work by blocking DNA repair mechanisms in cancer cells ("Science Now," Los Angeles Times, 12/19).
Richard Pazdur, who directs FDA's cancer drug division, said Lynparza "is an example of how a greater understanding of the underlying mechanisms of disease can lead to targeted, more personalized treatment" (Wall Street Journal, 12/19).
William Audeh, a geneticist and medical oncologist at Cedars-Sinai Medical Center's Samuel Oschin Cancer Institute, said the drug's approval is "really opening a whole new avenue of therapy. This drug is working in a fundamentally different way than chemotherapy: This is a cancer treatment that's been designed to hit this kind of inherited genetic weakness in the cancer itself."
According to Audeh, at least seven additional PARP inhibitors are being developed and tested for cancers that are associated with mutations of the BRCA gene and other cancers that could result from similar mutations ("Science Now," Los Angeles Times, 12/19).