UPDATED: AstraZeneca wins big with fast FDA OK of AZD9291 for lung cancer

AstraZeneca CEO Pascal Soriot

AstraZeneca ($AZN) gained a crucial accelerated FDA approval for its targeted lung cancer drug AZD9291 today, offering tangible evidence of the R&D turnaround CEO Pascal Soriot had promised investors several years ago. And now the drug, to be marketed as Tagrisso, will be put to the market test to see if it can live up to Soriot's $3 billion peak sales estimate while staring down a strong rival in late-stage development at Clovis Oncology ($CLVS).

The oral AZD9291 is targeted at non-small cell lung cancer patients whose tumors share a T790M EGFR mutation. The FDA's approval comes three months ahead of the PDUFA date, marking the agency's confidence in the data and its enthusiasm for targeted oncology therapies guided by a diagnostic test.

AstraZeneca won't be discussing the price of this new drug--a particularly hot topic these days--until next week. "Tagrisso price will be comparable to other oral cancer therapies and should be public early next week; we're not disclosing that today," a spokesperson tells FierceBiotech by e-mail.

The drug, which earned bragging rights and an inside regulatory track as a "breakthrough" therapy at the FDA, has long been one of AstraZeneca's top therapies in the pipeline. It has consistently demonstrated solid efficacy in clinical trials, recently demonstrating median progression free survival of 8.6 months in a midstage study.

That outcome--based on preliminary data that could yet change--fell short of the advantage that some analysts were looking for to keep competition from Clovis's rociletinib (CO-1686) at bay. And it was uncomfortably close to the 8-month PFS rate that Clovis had to explain for the same patients at the latest ASCO gathering in Chicago.

The treatment, though, remains a lead player in AstraZeneca's game plan for delivering more than $45 billion in annual revenue by 2023, a number cited when the pharma giant was fighting off Pfizer's ($PFE) megamerger attempt.

While AZD9291 loomed large at AstraZeneca, it was still overshadowed by the company's PD-L1 immuno-oncology drug durvalumab. That drug, though, may take considerably longer to get to the market. Soriot recently cautioned that with Merck ($MRK) and Bristol-Myers Squibb ($BMY) already on the market with PD-1 drugs, the FDA isn't likely to display the same kind of urgency that regulators put on display with today's approval. AstraZeneca has also experienced a few setbacks this year, including its decision to spin out the psoriasis drug brodalumab to Valeant ($VRX) after investigators tracked suicidal thinking among patients taking the drug. 

"Our understanding of the molecular basis of lung cancer and reasons these cancers become resistant to prior treatments is rapidly evolving," said Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "This approval provides a new treatment for patients who test positive for the EGFR resistance mutation, T790M, and is based on substantial evidence from clinical trials that shows Tagrisso had a significant effect on reducing tumor size in over half of patients who were treated."

- here's the FDA's release

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