CF in the News

I still need to post my usual Weekend Recap (late as usual), BUT…

Monday was a huge day for the CF community with the approval of Trikafta.  I actually receive emails from FDA because of my job so I saw “FDA approves new breakthrough therapy…cystic fibrosis” and was very confused.  I consider myself to be in the know regarding upcoming CF approvals and the triple combination therapy approval was targeted for early 2020.  March 2020 to be exact.  I think I read the email at least 10 times convinced this could not possibly be the news I’d been waiting for.  But it was.  And major kudos to Vertex because their application must have been on point - that was an incredibly fast approval.  This is the very first drug targeting CFTR function approved for A’s CF mutations.  I’m cautiously optimistic and really hopeful.  I’m so curious what will be discussed at A's next clinic appointment in November and if he'll be able to start taking the new drug soon.

Speaking of clinic, I never provided an update after A's last CF clinic appointment.  I was waiting until I learned his PFT numbers, but I still haven't seen them. A's clinic doesn't provide them at the appointment so he has to check them online and although I'm always super curious, he is NOT.  I do believe they were up slightly and in line with previous appointments.  I still wish they were higher and I'm not sure how much the antibiotics really helped.  If nothing else, maybe they prevented him from feeling worse.  A has also had a lot of GI issues since he was on the antibiotic.  We're not sure if there is a correlation, but he has an appointment with a GI doctor next month.