Tuesday, May 12, 2015

Great News from the FDA!

This afternoon, the FDA Advisory Panel recommended approval of the latest CF corrector drug, formerly VX-809/lumicaftor combined with Kalydeco, now known by it's official brand name, Orkambi! The final vote was 12 yes, 1 no. This is not the official and final approval, as the final word is expected to come on July 5th. But I learned today that the FDA uses the panel's recommendations to make their final decision, and they typically go with what the panel recommends. So while we still have to wait a couple months to find out if the portion of the CF population that carry 2 copies of DF508 and are ages 12 and older will soon have their magic pills in hand, we're significantly closer than we were before, and the remaining wait isn't much longer.

Orkambi's results weren't what we were hoping for, and during the studies it didn't show the improvements that Kalydeco had on patients with the G551 mutation. But there is enough improvement to slow the progression of the disease, which means it will buy more time for the double DF508 population, and Vertex can keep the momentum going on other corrector drugs like VX-661. And once this is approved, they can start the studies for the ages 6-12 group, and then the 2-6 group.

It's a bittersweet day, and July 5th will be bittersweet as well. If you've followed us for a while, you know I've talked about these drugs before, and have also mentioned that Judith only has 1 copy of DF508. Orkambi won't help Judith. The recommendation of approval, and (hopefully!) the final approval of Orkambi is another reminder that we're still waiting for our turn, waiting for our magic pills. All while we have to do whatever we can to keep her as healthy as possible as the disease slowly progresses. Our turn will come. I don't know when, but we'll get there. The waiting game absolutely stinks, but someday we'll join the ranks of CF patients who have a corrector drug that will benefit them.

At the same time, I'm rejoicing for my many friends whose loved ones will benefit from Orkambi, and any future correctors that are better! A chunk of my friends have children who are under the age of 12, so they won't have full access right away (but might be able to get it off-label), but it may not take long until the studies in the other 2 age groups are completed, and their children can start taking Orkambi.

I'd be lying if I said I wasn't tense today. I knew the panel recommendations were coming, but after a link to a live blog was posted in a group I participate in, I knew I'd be glued to my computer when I could. I literally let out a huge sigh of relief seeing the final vote results! Now to wait for the FDA's final decision...

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