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Carfilzomib Earns Strong Positive Vote From FDA Advisory Committee

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Published: Jun 20, 2012 4:21 pm

The Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA) has voted 11 to zero with one abstention in favor of car­filz­o­mib's bid to be approved as a new treat­ment for multiple myeloma.

Specifically, the majority of the committee voted that the risk-benefit assess­ment for carfilzomib (Kyprolis) is favorable for the treat­ment of patients with re­lapsed and refractory multiple myeloma who have received at least two prior lines of ther­apy.

Today's vote by the FDA committee went much better for car­filz­o­mib than had been expected.   A recent survey of major Wall Street investors, for example, showed a significant majority expecting a negative vote by the FDA panel (see related Beacon news).

In addi­tion, an FDA briefing document for today's meeting that was released earlier this week voiced strongly worded concerns about car­filz­o­mib's safety.  Publication of the FDA report caused the stock of Onyx Pharmaceuticals (NASDAQ:ONXX), the com­pany developing car­filz­o­mib, to drop more than 4 per­cent.

During today's meeting, however, the FDA staff presentation regarding car­filz­o­mib's efficacy and safety was considerably less critical of car­filz­o­mib.  In its discussion of potentially life-threatening side effects of the drug, for example, the FDA presentation noted that they occurred "with a low frequency" during relevant clinical trials.

In contrast, the presentations by Onyx employees and myeloma opinion leaders presenting on car­filz­o­mib's behalf laid out a strong case for both the efficacy and safety of the drug.

As a result, the momentum was in car­filz­o­mib's favor for most of today's meeting.  (See the Beacon's live coverage of today's meeting for details of all the presentations and question-and-answer sessions during the car­filz­o­mib review.)

Onyx completed an application to the FDA last September to have car­filz­o­mib as a new myeloma treat­ment (see related Beacon news).

The FDA will consider the advisory committee’s vote as well as comments made during today’s meeting when deciding whether to approve car­filz­o­mib's new drug application.

Although the FDA is not legally bound to follow its advisory committees’ recommendations regarding new drug applications, it usually does.

The FDA is required to make a decision about car­filz­o­mib’s application by July 27.  It also has the option, however, of extending this deadline if it feels it needs addi­tional time to complete its review.

For more in­­for­ma­tion about the FDA’s ongoing review of the car­filz­o­mib application, see the latest Beacon news about car­filz­o­mib.

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10 Comments »

  • suzierose said:

    YAY!!! .....on to FDA approval next!!

    Good news for MM patients!!

  • Terryl1 said:

    Carfilzomib has worked almost miraculously for me....knock on wood... and many others in the newly diagnosed trials, so I say a big YES!!!! This will help all afflicted my MM.
    Terry L.

  • gary said:

    Yes suzie rose. good news for the 75% of patients who can tolerate çarfilsomib.
    The rest of us can only hope that the FDA will recommend
    that onyx use the available tools in the personalized medicine
    Arsenal to getting the dose individualized so we can benefit from
    Carfilzomib as well.

  • suzierose said:

    I agree GAry!!

    We need every tool (drug) in the box, to manage this disease. For every person it helps it is such a wonderful thing, even if some are helped more than others. No one drug is a panacea. We do have to be informed and make good choices. But thank the lord, we have choices!!

    ..praying for even MORE choices...

  • Stan said:

    Great news! Now I will work on spelling it... carfilozeb...carfilozomib carfilzomib.
    Krypolis...sort of sounds like "cripple us". I'll stick with carfilzomib.

    Since Velcade didn't do much for me, is it true that carfilzomib has a lower chance of working for me?
    Either way, I'm happy for the MM community.

  • suzierose said:

    hehehehe Stan!

    you 2 funneee! "cripple us"...lol lol

    Carfilzomib could work since it works at a different site...know the safety profile...and try it and see. IF the FDA approves it...we not HOME yet!!..this was just advisory cmte

  • tiziano said:

    Hi guys. Happy for your excitement, but difficult to share it.

    I tend to say the the real problem for our desease is not the front line therapy but the therapy for relapsed/refractory patients. So ok we have undoubtedly got a new tool (many thanks to the researchers for their unvaluable effort), but no big change (am I wrong ?) for relapse/refractoriness (22% of overall response, ?together with Dex).

  • suzierose said:

    Hi Tiziano,

    I agree that 22% is far from stellar. Unfortunately, that is the up-to-date progress for MM...I too wish it was far better. Nevertheless, it is good for all MM patients to have more choices,no?

    Even with the 22% some relapsed/refractory patients will be able to delay disease progression, perhaps until another agent becomes available. One thing I have noticed about some of the clinical trials in the RR patient population is that some have had 5 prior therapies. That offers hope as despite prior failure those patients were still able to manage their disease and continue on to a different therapy.

    The choices with myeloma are just not easy. I wish they were.

  • Ron Harvot said:

    Also remember that when Velcade was approved it only had a 27% overall response in the RR environment. The 22% response rate for Carfilzomib includes patients that stopped responding to Velcade.

    Later tests using Velcade combined with Revlimid and Dex in newely diagonsed patients showed an almost 100% response rate.

    The use of Carfilzomb is being sought for RR patients that have aleady gone through 2 other lines of treatment.

    To me this is great and exciting news. Now on to getting Pomalidomide approved!

    Ron

  • tiziano said:

    Thank you for underlyning the meaning of that 22%. I agreed that it gives us more chance. Maybe I was expecting more as I had been hearing about Carfilzomib for a few years already. I remain not excited but I agree this is a hard achievement against myeloma and that today is time for celebrating.