Roche's Perjeta nabs speedy FDA nod in early breast cancer

It's official: Perjeta is now the first cancer drug approved to treat patients before surgery. Developed by Roche's ($RHHBY) Genentech unit and already approved for women with advanced HER2-positive breast cancer, Perjeta can now reach a huge new group of patients at early stages of the disease.

The approval is a coup for Roche, which is developing its new generation of HER2-fighting drugs in advance of biosimilar competition for its gold-standard therapy, Herceptin. It's also a landmark for the FDA, which loosened up cancer drug approval guidelines to speed new therapies to market. Not only did the FDA's blessing come a full month before the decision was actually due, but Perjeta is also the first drug to qualify for approval based on tumor shrinkage rather than survival data.

And shrink them it did. In one midstage trial, almost 40% of patients treated with Perjeta, Herceptin and the chemo drug docetaxel saw their tumors shrink to undetectable levels. In a companion safety study, Perjeta's side effects in early-stage patients proved consistent with those seen in patients with advanced disease.

"A new approval pathway has made Perjeta available to people with HER2-positive early breast cancer several years earlier than previously possible," Genentech chief medical officer Dr. Hal Barron said in a statement. "Together with the FDA, we've charted new territory. We look forward to working with health authorities around the world to explore additional ways to bring promising medicines to patients more quickly."

The presurgery treatment won't be cheap; Perjeta plus Herceptin is expected to run $27,000 to $49,000 for 9 to 18 weeks' worth of therapy. It won't take the place of postsurgery drug therapy. Some 15,000 patients could be eligible for treatment before surgery each year, making the new use a potentially lucrative one for Roche. Launched in June 2012, Perjeta was instantly pegged as blockbuster material, with some analysts predicting mega-status at $8.5 billion in peak sales.

Perjeta is intended for use in tandem with Herceptin--or with Herceptin's brand-new follow-up, Kadcyla, which uses new technology to deliver Herceptin's active ingredient directly to tumors. Roche hopes that Kadcyla's results will trump Herceptin's, allowing the new drug to take over, sales-wise, as biosimilars leach away Herceptin revenues. The company continues to build up its case, too: In data presented at the European Cancer Congress over the weekend, Kadcyla beat both Herceptin and GlaxoSmithKline ($GSK) rival Tyverb at keeping cancer growth at bay.

- read the FDA announcement

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