Portola shoots for early FDA approval after nabbing 'breakthrough' status

South San Francisco-based Portola Pharmaceuticals ($PTLA) has nabbed boasting rights to the FDA's new breakthrough drug designation for andexanet alfa (PRT4445), its Factor Xa inhibitor antidote designed to hit the brake on blood thinners when they turn dangerous. And the biotech says that the BTD helps set the stage for its campaign to win an accelerated approval while pursuing a 2014 launch of a registration trial.

This antidote to anticoagulants is meant to complement Portola's oral Factor Xa inhibitor betrixaban, now in a late-stage study for venous thromboembolism prevention in acute medically ill patients. And its effect is being studied on Eliquis, Xarelto, Lovenox and Lixiana.

Portola joined the big biotech rush to go public earlier this year, enjoying one of the more successful IPOs in the class of 2013 as investors bought in, encouraged by backing from Pfizer ($PFE), Johnson & Johnson ($JNJ), Bristol-Myers Squibb ($BMY) and Bayer. And FierceBiotech named the company to the Fierce 15 back in 2009.

The jury is still out on just how significant the FDA's BTD project will be for drug developers. The cancer division at the agency has started handing out some early approvals, cutting months off the application process for J&J's ibrutinib. But there have yet to be any significantly accelerated OKs. The FDA is promising to maintain an open door policy for these therapies, offering advice and guidance on speeding up the trial and review process.

"The FDA's decision to designate andexanet alfa as a breakthrough therapy reaffirms the urgent need for an antidote to Factor Xa inhibitors, and we believe it demonstrates that andexanet alfa's properties and data distinguish it from currently used agents or others in development," said William Lis, Portola's chief executive officer. "Pro-coagulant agents being used in the absence of an antidote are unproven, have limited biological rationale for their effectiveness to reverse Factor Xa inhibitors, and have been shown to cause serious blood clots. Therefore, andexanet alfa marks an important advance in the field, and our goal is to bring it to market as quickly as possible."

- here's the press release

Special Report: The top 10 biotech IPOs of 2013 - Portola Pharmaceuticals

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