Despite loud objections from its advisers, the FDA granted an accelerated approval to AstraZeneca's ($AZN) ovarian cancer treatment, clearing an oral therapy the U.K. drugmaker believes will bring in blockbuster sales.
The drug, olaparib, is designed to treat advanced ovarian cancer in women with defective BRCA genes, and the FDA's approval covers patients who have already failed on three or more chemotherapy treatments. The nod comes just one day after the European Medicines Agency gave the drug its blessing, making olaparib the first poly ADP-ribose polymerase (PARP) inhibitor cleared in the U.S. AstraZeneca expects the treatment, to be marketed as Lynparza, to bring in $2 billion a year at its peak.
The FDA's approval is based on Phase II data in which the drug charted a 34% objective response rate for an average of 7.9 months. Treatment with olaparib also led to progression-free survival (PFS) of 11.2 months versus 4.3 months on placebo.
Those same results weren't enough to convince a panel of FDA advisers over the summer. In June, the agency's cancer drug committee picked apart olaparib's potential, taking issue with its failure to significantly improve overall survival, pointing to a few alarming adverse events and expressing doubts in the reliability of its PFS data. The group voted 11-2 against recommending an early approval for the drug, asking the agency to wait out AstraZeneca's ongoing Phase III study on olaparib before green-lighting it.
But AstraZeneca came back with additional information supporting the drug's use in thrice-failed patients who carry BRCA mutations, the FDA said, which was apparently enough to sway the agency's oncology division, affirming cancer chief Richard Pazdur's penchant for hurrying along new treatments that could change the standard of care.
Olaparib is approved alongside a companion diagnostic from Myriad Genetics ($MYGN) that can suss out which patients have the right genetic profile for the drug.
"Today's approval constitutes the first of a new class of drugs for treating ovarian cancer," Pazdur said in a statement. "Lynparza is approved for patients with specific abnormalities in the BRCA gene and is an example of how a greater understanding of the underlying mechanisms of disease can lead to targeted, more personalized treatment."
AstraZeneca unveiled its $2 billion expectation for olaparib earlier this year when the U.K. drugmaker was fending off unwelcome advances from Pfizer ($PFE). AstraZeneca has even higher hopes for MEDI4736, a PD-L1 therapy the company believes will top out at $6.5 billion a year; and AZD9291, a Phase I lung cancer treatment tabbed to peak at $3 billion.
For each of AstraZeneca's oncology contenders, reaching those lofty goals will depend on success across a range of cancers. For olaparib, the company is running concurrent Phase III trials in ovarian, gastric and breast cancers, hoping to win a string of approvals and cobble together a blockbuster.
- read the statement