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Thread: Kalydeco gets approval for eight more mutations

  1. #1

    Kalydeco gets approval for eight more mutations

    Good news for approx. 150 people with CF 6 and older.

    a supplemental New Drug Application (sNDA) for KALYDECO
    TM (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have one of eight additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. KALYDECO was first approved in January 2012 for people with CF ages 6 and older who have at least one copy of the G551D mutation. With the approval of the sNDA, KALYDECO is now approved for use in people with CF with the following nine mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. In the United States , approximately 150 people ages 6 and older have one of the additional eight mutations for which KALYDECO is now approved.

  2. #2
    welshwitch
    Guest
    Wow! Great news!!!

  3. #3
    Senior Member
    Join Date
    Oct 2007
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    So happy for those 150 people!

  4. #4
    Hopefully that means it will get approved for more later on too.

  5. #5
    Junior Member
    Join Date
    Mar 2012
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    I totally think my son is one of those 150 people - I about drained every ounce of liquid in my body with tears when I talked to his pulmonologist about this. Of course I was the one who had to inform HIM - but I don't really care. Does anyone know anyone who is on this? Does it mean an end to the enzymes and lung therapy? That would just be the best news ever if it does!

  6. #6
    Senior Member
    Join Date
    Aug 2006
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    Mom24dodd: My 2 daughters with G551D (as one mutation) have been on Kalydeco for 2 years. They have not stopped any routine meds including enzymes. What it has done is make them more stable and neither had needed hospitalizations since. My one daughter was in the hospital every few months for several years prior to that, so we credit the Kalydeco. They both stopped taking oral antibiotics all the time too. PFTs were good prior and are staying good.

    I know some have said they could stop enzymes and back way off on breathing treatments, but that hasn't been our experience.

    This is still AMAZING news that your CFer can get this groundbreaking drug! I hope you'll see a huge improvement.
    Mom to two daughters with CF, G551D/2622+1G>A

  7. #7
    Junior Member
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    Mar 2012
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    2roses: Wow - ok - seems like your results are very good. My son will be 9 this year and has only had to be on anti-biotic one time - thankfully - and never has been hospitalized for being sick. He has autism too so we really cannot do PFTs - we just get the respiratory therapist to do deep throat culture and every so ofter we get bronchoscopy done.
    Thanks so much for your input!

  8. #8
    Super Moderator
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    Mar 2012
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    2,035
    Mom24dodd this blog gives you another before after look and might be what your son could experience given his current status...no treatments would be heaven, but even no hospitalization is an amazing thing...so happy for all benefitting from K!
    http://luckycfmom.blogspot.com/

  9. #9
    Junior Member
    Join Date
    Aug 2008
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    2
    Aboveallislove: thank you for the blog link from "luckycfmom". I was so inspired and moved with pure joy at reading her son's results. I'm with the many,waiting with great anticipation, for the combination of Kalydeco and VX809 to complete trials (expected in June 2014) and get FDA approval (expected December 2014). We all need to keep supporting the CF Foundation.

  10. #10
    Allansarmy
    Guest
    This is exciting news indeed. 150 people is 150 people who do not have to suffer as much or possibly even extend their lives longer. Love to hear news like this. Not to sound too self indulgent but any news on the Delta mutation which if I am not mistaken is the most popular of all mutations for CF? Also, if a person has the double Delta mutation like my son, does that change things with this drug if it gets approved. Or I should say "WHEN" I must stay positive.

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