Thursday, July 2, 2015

Orkambi Is Approved!

It's a joyous, and also a bittersweet, day in the CF community again. Today, the FDA approved the latest corrector drug, Orkambi, for CF patients 12 and older with 2 copies of the F508del mutation!

This is the outcome we were expecting, and yet still hoping for, after the affirmative nod from the panel a few months ago.

Now that it's approved for the 12 and older set, they can start the trials in the 6-12 set, and after that, 2-6. It's an exciting time for sure!

Just like the emotions I had a few months ago, I can't help but feel a bit of sadness for Judith, and everyone else who is still waiting for our turn. It will come, but how long are we going to have to wait? We're getting closer, and now more than ever, I'm feeling more of an urgency to keep her as healthy as absolutely possible so she can get as much of the benefits from the drugs that she can.

Still, I rejoice with the rest of the community. I am happy for my friends whose loved one will be able to start taking this drug now, and for those who will be able to start this drug once the trials for the younger age groups are completed and approved.

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