AstraZeneca eyes $2B in sales with FDA nod for ovarian cancer fighter Lynparza

Briggs Morrison, AstraZeneca EVP, global medicines development and chief medical officer

AstraZeneca ($AZN) won accelerated FDA approval for its ovarian cancer fighter Lynparza (olaparib), opening the door for potential blockbuster sales of the drug and helping the company distinguish itself from rivals in the BRCA playing field.

Lynparza belongs to a new class of treatments, poly ADP-ribose polymerase (PARP) inhibitors, which are designed to zero in on cancer cells and leave a patient's normal cells untouched. The drug snagged a regulatory signoff from the European Medicines Agency one day before its stateside approval, making it the first drug cleared in the U.S. to treat advanced ovarian cancer in women with defective BRCA genes. AstraZeneca expects Lynparza to bring in $2 billion a year in peak sales.

Briggs Morrison, AstraZeneca's chief medical officer, called Lynparza "an excellent example" of next-generation R&D into targeted medicines and "a much-needed" new option for patients with ovarian cancer. And AstraZeneca has even higher hopes for Lynparza down the road. "Today's approval also marks the first of what we hope will be a number of indications in which this medicine has the potential to improve the lives of cancer patients," Morrison said.

The FDA based its approval on a Phase II study comparing Lynparza to a placebo in BRCA-positive patients who'd relapsed after three rounds of chemotherapy. In the 137-patient study, the drug chalked up a 34% response rate, with half of patients responding for at least 7.9 months. The drug was approved alongside a companion diagnostic, BRACAnalysis CDx, from Myriad Genetics.

Regulators were not always rosy about Lynparza's prospects. Over the summer, an FDA panel voted 11-to-2 against early approval, citing questions about potential side effects and uncertainties about progression-free survival data. AstraZeneca bounced back with additional information showing Lynparza's success in treating patients who have failed chemotherapy three times and clearing the way for EMA and FDA approval.

The regulatory signoffs come at a critical moment for AstraZeneca, as the company touts its status as a solo act and beefs up its cancer drug portfolio. The U.K. drugmaker contends that Lynparza can bring in $2 billion a year, a figure it cited when fending off Pfizer's unwelcome pursuit, but it faces stiff competition from AbbVie ($ABBV) and Clovis Oncology ($CLVS), which are forging ahead with their own BRCA therapies. AstraZeneca appraises its overall oncology pipeline at $12 billion.

Meanwhile, AstraZeneca is testing Lynparza in a variety of cancers. In November, the Institute of Cancer Research's Johann de Bono touted promising results from an early clinical trial in prostate cancer. Late-stage studies are eyeing Lynparza in breast cancer, gastric cancer and pancreatic cancer. And in ovarian cancer, AstraZeneca has three Phase III studies underway, evaluating Lynparza as a treatment for relapsed patients and as a maintenance therapy.

- read AstraZeneca's statement
- here's the FDA's statement
- and here's FierceBiotech's take

Special Report: The top 10 pharma companies by 2013 revenue - AstraZeneca