FDA stokes blockbuster hopes with 'breakthrough' brand for Pfizer cancer drug

The FDA has endorsed Pfizer's ($PFE) high hopes for the breast cancer drug palbociclib (or PD-0332991), handing out its coveted "breakthrough" drug status after posting some impressive interim results from a mid-stage study tracking progression-free survival among breast cancer patients.

Palbociclib, which Pfizer CEO Ian Read recently singled out as one of the pharma giant's hottest Phase III assets, is an oral inhibitor of cyclin-dependent kinases (CDK) 4 and 6. Last December investigators said that they had tracked PFS of 18 months in a Phase II study, triggering some blockbuster dreams among some of the analysts following the company.

The drug represents a key exhibit in Pfizer's case that its R&D group, once an industry poster child for dysfunction and failure, had turned an important corner. Last year Pfizer picked up a record 5 FDA approvals, including OKs for new first-line therapies like Eliquis and Xeljanz. And the turnaround occurred as the company completed a jarring downsizing in R&D, retreating in some areas but staying focused on cancer as one of its primary R&D categories. 

The designation also helps clarify the FDA's thinking on its new breakthrough status. Pfizer now joins some of the industry's top players, companies like Johnson & Johnson (which now has three of these after the FDA tapped the cancer drug ibrutinib), Vertex (VX-809 and Kalydeco for cystic fibrosis) and Novartis (LDK378 for lung cancer).

The agency intends to help these companies speed these drugs through the development process, offering them an inside track in an effort to selectively accelerate approvals for the most promising drugs in the pipeline. Its early days yet, but the FDA so far has demonstrated its preference for marquee industry drug programs from some of the best-known R&D operations around the globe. Whether there's any significant room for small biotech companies without the same deep pockets and lengthy track records is yet to be determined conclusively, but the early returns would suggest otherwise.

Pfizer's success here could add some luster to other programs following the same pathway. A story in Nature counted Lilly's ($LLY) LY2835219 and Novartis' ($NVS) LEE011 among the drugs with a similar CDK-inhibitor profile, offering a shot at blocking cancer cell proliferation.

"We appreciate the opportunity that Breakthrough Therapy designation provides to work closely with the FDA on the development of palbociclib," said Dr. Mace Rothenberg, senior vice president of clinical development and medical affairs for Pfizer's oncology business unit. "Palbociclib is one example of Pfizer's commitment to identifying and translating innovative science into meaningful new treatment options for cancer patients."

- here's the press release

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