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  • 1.  Oncology small molecule clinical development advice

    Posted 13-Oct-2020 19:02
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    Hi RAPS Community,

    A small molecule is currently under development for oncology. No IND opened yet. The plan is to conduct a monotherapy clinical study with this compound and extend it to the US. Currently, a Phase II combination study in EU is ongoing with this molecule. 

    Do you know if this is absolutely necessary to have monotherapy clinical data for registration in the US, or if we can consider using the combination data? 

    Thank you,

    Best Regards,




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    Charlene Revel
    Senior Regulatory Scientist
    Cambridge MA
    United States
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  • 2.  RE: Oncology small molecule clinical development advice

    Posted 15-Oct-2020 12:26
    No replies, thread closed.
    "Currently, a Phase II combination study in EU is ongoing with this molecule."

    Is this combination with an approved drug? If so, other than preliminary phase 1 data from monotherapy (either from EU or both US&EU centers ....), one could pursue combination (add on) therapy in a registration trial if your currently ongoing Phase II in the EU  is promising with registration worthy data.

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    GRSAOnline
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    Original Message


  • 3.  RE: Oncology small molecule clinical development advice

    Posted 16-Oct-2020 05:57
    No replies, thread closed.
    Hi Charlene,

    FDA will generally require monotherapy data to demonstrate the activity (or lack of) of the compound itself to support registration in combination with another therapy to allow FDA to assess the benefit of each component of the combination.  An exception to this might be if the mechanism of action of your compound only works in synergy with another compound.  But even in this case, FDA would likely want safety data on your compound alone.

    As always, meet with FDA early and discuss / align on the development path for your compound, especially to define how much monotherapy data FDA may require before agreeing to studying the combination in the US.

    Good luck!

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    Mark De Rosch PhD FRAPS
    Chief Regulatory Officer
    Epizyme, Inc.
    Cambridge, MA
    United States
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    Original Message


  • 4.  RE: Oncology small molecule clinical development advice

    Posted 16-Oct-2020 14:38
    No replies, thread closed.

    Hi Mark,

    Thank you for sharing your thoughts on this topic. This is quite helpful and confirm the plan to move with some monotherapy data first with the single agent. This will be discussed during the preIND meeting to make sure we are aligned with the FDA.

    Best Regards,
    Charlene 



    ------------------------------
    Charlene Revel
    Senior Regulatory Scientist
    Cambridge MA
    United States
    ------------------------------

    Original Message


  • 5.  RE: Oncology small molecule clinical development advice

    Posted 16-Oct-2020 14:36
    No replies, thread closed.

    Hi Narayan,

    Thank you for your feedback. Indeed, the combination is with an approved drug and the goal is to pursue combination in a registration trial once we will get monotherapy data. 

    Kind Regards. 
    Charlene 



    ------------------------------
    Charlene Revel
    Senior Regulatory Scientist
    Cambridge MA
    United States
    ------------------------------

    Original Message


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