If You’ve Got a Real Breakthrough, the FDA Wants To Talk

1/14/13Follow @xconomy

If you ever want to have an interesting chat with a healthcare journalist, ask about use of the word “breakthrough.” Like many reporters, I generally avoid it. The word may be good for generating eyeballs/pageviews/ratings, but it’s usually an exaggeration that serves the financial interest of drugmakers and gives patients false hope.

Sometimes, though, breakthroughs do occur. And now there’s a federal law on the books that’s encouraging companies to develop more groundbreaking, innovative therapies. From now on, you can expect to see drugmakers talk about getting official “Breakthrough Therapy Designation” from the FDA, and it won’t be just an empty, boastful phrase. It’s still early in the game, but I expect this new regulatory incentive structure will tilt the drug industry’s priorities toward difference-making therapies, and away from incremental advances that have prevailed for the past 20 years.

This past week, Cambridge, MA-based Vertex Pharmaceuticals (NASDAQ: VRTX) raised awareness of the new regulatory meaning for the word “breakthrough” when it said its cystic fibrosis treatments ivacaftor (Kalydeco) and VX-809 were granted the first two “Breakthrough Therapy Designations” by the FDA. The company was conservative in its statement, saying that the implications of the action “cannot be determined at this time.” Vertex CEO Jeff Leiden shared a little more with Bloomberg News, saying, “it’s an opportunity to have a different set of interactions and collaborative discussions” with regulators.

That might not sound like much, but there’s more. The FDA was instructed to prioritize reviews of such “breakthrough” therapies through the Food and Drug Administration Safety and Innovation Act (FDASIA) that was signed into law in July. For policy wonks, this is tied to the fifth extension of the Prescription Drug User Fee Act. Here’s a key section of the law (Section 902), regarding development of breakthrough drugs:

“The Secretary shall, at the request of the sponsor of a drug, expedite the development and review of such drug if the drug is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.”

What does that really mean? I asked a lot of biotech pros that question last week at the JP Morgan Healthcare Conference in San Francisco. I got more than a few shrugged shoulders. But one clear answer came from Tom Hughes, the CEO of Cambridge, MA-based Zafgen, a venture-backed startup working on a novel drug for severe obesity.

Tom Hughes, CEO of Zafgen

Hughes has been encouraged by public statements from Janet Woodcock, a top FDA official, and what he reads in the new law. Woodcock has made repeated comments in public about the need to encourage more “breakthrough” drugs.

Check this comment from Woodcock on an FDA blog last month:

 “The new law is designed to get “breakthrough” therapies developed as quickly and safely as possible so they can be available to treat the patients who need them. We’re excited about it! In fact, although the law is only a few months old, we’re already putting it to use. Recently we identified the first therapy to receive this special designation. And it likely won’t be long before we have more. Several other drug developers have already made inquiries and there is lots of interest in the pharmaceutical industry in taking advantage of this new development tool.”

Hughes, a former Novartis research leader, says he sees an opportunity for Zafgen’s beloranib to get one of these new designations. The company has shown that the injectable can help seriously obese people lose a lot of weight, quickly, in small clinical trials.

I’ll leave the value judgments to the FDA about whether Zafgen’s drug should count as a “breakthough therapy” in need of a shorter/faster clinical development path. But Hughes says he’s looking forward to submitting an application to be reviewed by the FDA’s Office of New Drugs, led by John Jenkins, one of the agency’s high-level officials.

If the agency decides within its 60-day review period to grant this designation, then Zafgen’s application would zoom up on the FDA’s priority list. Normally the company would wait weeks or months to hear feedback about a clinical trial plan from overextended FDA reviewers. Instead, the company could get … Next Page »

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  • Rogan

    How long until only politically connected firms get “breakthrough” status? I could see a possible future where only drugs being bet on by Wall Street get a bump and everything else gets slowed down.