FDA panel gives 'historic' thumbs-up to Roche's Perjeta in early breast cancer

You might call Perjeta a guinea pig. The new breast cancer drug from Roche's Genentech unit could soon become the first approved for cancer patients before they've had surgery. It would also be the first approved under new FDA guidelines for speeding cancer drugs to patients with early-stage disease.

Perjeta nabbed an FDA panel's recommendation for neoadjuvant use, or treatment before surgery, in patients with HER2-positive breast cancer. And it won that recommendation just a little over a year after its original launch for late-stage, HER2 disease. That's truly unprecedented; as Bloomberg notes, Roche's ($RHHBY) HER2 gold-standard drug Herceptin won its original approval in late-stage breast cancer in 1998, and wasn't approved for early-stage disease until 2006.

Perjeta could make that seven-league leap because of new FDA guidelines allowing tumor shrinkage--rather than survival data--to be a study endpoint. The move was designed to do exactly this: get cutting-edge treatments to early-stage patients in hopes of stopping the disease in its tracks. Drugs could win accelerated approval on the basis of short-term studies, with long-term survival data to follow from post-marketing trials.

Genentech's R&D chief Hal Barron

The committee's recommendation "is a step toward bringing Perjeta to people with HER2-positive early stage breast cancer, where treatment can potentially prevent the disease from returning and spreading," said Genentech's R&D chief Hal Barron.

Perjeta's new indication would be based on data from two short, small mid-stage trials. In the Neosphere study, which measured efficacy, 39.3% of patients treated for 12 weeks with Perjeta, Herceptin and docetaxel chemotherapy saw a complete pathologic response, which means traces of tumor tissue were undetectable. In the Herceptin-plus-chemo arm, 21.5% enjoyed the same result. The Tryphaena cardiac safety study was smaller--225 patients--but it suggested one important thing. Side effects were consistent with earlier Perjeta trials in metastatic patients.

"This is a historic moment," Cleveland Clinic professor Dr. Mikkael Sekeres, who chaired the panel, said after the vote (as quoted by the New York Times). Richard Pazdur, the FDA's oncology chief, called it a "quantum leap."

So, just as with any prodigy, Perjeta will be watched carefully. How will the drug perform in the real world? Will the survival data from a late-stage trial, due in 2016, prove that neoadjuvant treatment actually extends patients' lives? Does the Perjeta case herald a series of FDA nods for earlier use of cancer drugs?

The agency will issue its final decision by October 31, but if the FDA staff's review is any indication, the approval is all but a done deal. Roche may soon be back for more; it's also studying Kadcyla, the much-anticipated "Herceptin-plus" treatment, for neoadjuvant use.

The early treatment wouldn't be cheap: Genentech tells the NYT that using Herceptin and Perjeta for 9 to 18 weeks before surgery would cost $27,000 to $49,000. Neoadjuvant drug therapy wouldn't replace drug treatment after surgery.

It could be quite lucrative for Roche, with an estimated 15,000 patients eligible for neoadjuvant treatment each year. And Perjeta has already racked up impressive sales for a new treatment. For the first half of this year, it brought in 108 million Swiss francs, or about $116 million. Analysts figure it could hit $1.6 billion by 2016.

- see the Genetech release
- read the Bloomberg piece
- get more from the NYT

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