Hopeful for FDA approval of Orkambi tomorrow

Aboveallislove

Super Moderator
The target date for approval is July 5, which is a Sunday and with Friday seeing most (all?) federal government offices closed in celebration of Independence Day, it's very likely the FDA will announce approval of Orkambi tomorrow. The July 5 date, though, isn't had and fast...but here's hoping! Watch for an 8 a.m. or 4 p.m. announcement (before/after market)
 

Aboveallislove

Super Moderator
Yes! 2 copies of delta f508. It would be approved for 12 and older but if close to 12 (and especially if at the weight they used for the trial), the doctor might do off label. DS just turned 6 so not yet covered. The 6 - 11 study is fully enrolled and should have results in October. Not sure how long the FDA will take to expand label, but once results are out assuming it is all okay, I'll then push for off label. The 6-11 is only for drug exposure (matching the levels in the blood stream as for older) and safety...in U.S. the FDA is not requiring proof for 6-11 it "works". I'll post as soon as anything is released.
 

Rebjane

Super Moderator
My daughter is 12 but only has one copy of delta F508..I've wondering if is would be used in such a case..
 

Aboveallislove

Super Moderator
It would only be fda approve for those with two copies. Is her other a class 1 mutation? Can you post it and ill see if any evidence on how the drug might work....also do you know of the 661 trials for those in her situation!
 

Ratatosk

Administrator
Staff member
Eagerly awaiting as well. DS is 12. Doctor said to call his office when (if) approved and he'll write out a script. Based on friends whose kids are on Kalydeco, I'm assuming we'll have to go thru a specific pharmacy rather than our local one.
 

triples15

Super Moderator
The target date for approval is July 5, which is a Sunday and with Friday seeing most (all?) federal government offices closed in celebration of Independence Day, it's very likely the FDA will announce approval of Orkambi tomorrow. The July 5 date, though, isn't had and fast...but here's hoping! Watch for an 8 a.m. or 4 p.m. announcement (before/after market)

Whoo hoo! Thanks for the heads up, I hadn't even thought about the fact that the 5th is a SUNDAY. I'll be watching!
 

Ratatosk

Administrator
Staff member
A friend of mine who attended a CFF advocacy mtg last spring heard that there's another drug specifically for ddf508 coming down the pipe, too that'll blow Orkambi out of the water.
 
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Allansarmy

Guest
I want to keep this thread positive as my 19 year old son has ddF508. He has been through so much. My only question is why am I finding the negative articles that are sceptical at best for Orkambi? Hate waiting but what can you do. I will fight for him to get this drug but why do they put doubt out there in their studies?
 

Aboveallislove

Super Moderator
Ratatosk: YES! there are so many things in the pipeline that will run circles around Orkambi (or maybe with Orkambi). Do you know which specific drug they were talking about? I'd love to stalk that too!!!

Allansarmy: I think it depends on the source/focus of the article. There is a lot by investors who look at it from a $ standpoint only and they are trying to objectively evaluate the drug. For competitors, they are trying to boast their own research. But in short, I think it's because Orkambi isn't Kalydeco. It's results are "modest" and not the miracle of Kalydeco. But for some it might be and for others modest will be huge in just day to day life. I often think of living with CF as walking on our train track of life, never knowing when the big black train will come to get us. Usually when it comes, we've already set our children safely off on their own tracks, but with CF their track isn't finished and we hear that dam# whistle all the time as we watch the builders trying to fix the broken tracks. I think of Orkambi as another length of track or a siderail on which they can wait while the workers fix their track and silence that whistle. But for know all I want is a little something. Maybe a little less chance of an exacerbation or colonization. Or fewer GI issues. Or less pain when DS takes a bath. Hugs dad and hoping you son is one for whom it is a miracle!
 
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