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Thread: Guardedly EXCITED??!

  1. #1

    Guardedly EXCITED??!

    https://www.cff.org/News/News-Archiv...stic-Fibrosis/
    Does this mean what I think it means?
    That some heterozygotes can try the new triple combination?????????????

  2. #2
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    ToriMom-

    The drug this news article is referring to isn't the triple combo. This new drug (Smydeko) will treat those with DDF508 and it will also treat heterzygotes that have one DF508 plus select residual function mutations. It's from the 661-108 and 661-114 studies.

    If I remember right, your daughter has df508 + minimal function mutation, which Smydeko (Tezafactor/Ivacaftor) will not be appoved for.

    But stay excited because there was also a news release on January 31st that referred to the triple combo.
    I'm not sure if you heard of the incredible triple combo results. Hopefully you'll be able to get her into one of the upcoming Phase 3 studies!! Best of luck to you. The studies haven't opened yet. I'll post again when they have!



    The following info. is from Phase 2 triple combo studies (not Tezacaftor/Ivacaftor).

    1/31/2018 FROM CFF.ORG:

    The Phase 2 studies for VX-659 and VX-445, for which early results were announced today, examined the effectiveness of each compound in combination with tezacaftor and ivacaftor in individuals with one F508del CFTR mutation and one minimal function mutation
    . Participants in these clinical trials who took the highest dosage of VX-659 and VX-445 as part of a triple combination saw improvements in their FEV1
    by an average of 13.3 percent and 13.8 percent respectively after four weeks of treatment. Both groups also experienced significant improvements in their sweat chloride levels and their quality of life measurements.

    “The research released today underscores the potential of next-generation combination therapies to be significantly more effective than current modulators,” said Michael Boyle, M.D., senior vice president for therapeutics development for the Cystic Fibrosis Foundation. “Importantly, the ability of these potential drugs to treat individuals with a single F508del mutation means that more people than ever before could benefit. This is very exciting news for our community, and we are grateful to those who participated in the clinical trials that made today's announcement possible.”

    Vertex Pharmaceuticals Inc. anticipates that the first of the Phase 3 clinical trials for VX-659 in combination with tezacaftor and ivacaftor will begin enrollment in the first half of 2018. The studies will be conducted in people with one F508del mutation and one minimal function mutation, as well as in people with two F508del mutations. Vertex also plans to begin a Phase 3 clinical trial for VX-445 in combination with tezacaftor and the compound VX-561 as a once-daily therapy in people with one F508del and one minimal function mutation, and in people with two F508del mutations, in mid-2018.

    “Today's announcement highlights the tremendous potential of the triple combination therapy to address the underlying cause of the disease in more people with CF than ever before,” said Preston W. Campbell, III, M.D., president and CEO of the Cystic Fibrosis Foundation. “We are extremely excited by the prospect of reaching over 90 percent of the CF population with these life-saving modulators and are committed to ensuring that all people with CF will one day have disease-modifying treatments.”

    The Cystic Fibrosis Foundation provided $150 million in research funding to Vertex to support its pursuit of treatments that address the underlying defect in CF, including $75 million to develop next-generation candidates currently in clinical development.

    Summary of Results:
    In the early results of the Phase 2 clinical trial of VX-659 in combination with tezacaftor and ivacaftor, the 63 participants in the study with a single F508del mutation who received the highest dose of VX-659 had a 13.3 percent increase in lung function and a decrease in sweat chloride of 51.4 after four weeks of treatment. Improvement in their quality of life score (CFQ-R) was 24.6 points.
    In the Phase 2 clinical trial of VX-445 in combination with tezacaftor and ivacaftor, the 65 participants in the study with a single F508del mutation who received the highest dose of VX-445 had a 13.8 percent increase in lung function and a decrease in sweat chloride of 39.1 after four weeks of treatment. Improvement in their CFQ-R score was 25.7 points.
    Last edited by jricci; 02-13-2018 at 10:33 AM.
    45 y/o with CF (R334W and DF508)

  3. #3
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    I was excited too thinking it was the triple combo; however, it's still another tool in the toolbox, helps some other mutations who are heterozygous delta f508. One step closer in my opinion!
    Parent to a child wcf double delta f508

  4. #4
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    Quote Originally Posted by Ratatosk View Post
    I was excited too thinking it was the triple combo; however, it's still another tool in the toolbox, helps some other mutations who are heterozygous delta f508. One step closer in my opinion!
    Absolutely
    I think it will be a game changer for many--especially those with DDF508 that weren’t able to tolerate Orkambi’s side-effects.
    It was also shown to be more effective compared to Kalydeco for those with DF508+some residual function mutations.
    Not only a new tool, but an improved tool. Very exciting!
    45 y/o with CF (R334W and DF508)

  5. #5
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    Does anyone know how soon we can get Symdeko? My Vertex case manager emailed me yesterday to let me know that my children’s mutation would be a candidate for Symdeko. They are currently on Kalydeco. I contacted our CF doctor, and the nurse said that they were waiting on their Vertex rep to meet with them on more info and how to handle insurance companies. Just curious if anyone has heard from their Dr on when this new promising medication can be prescribed?

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    I know I saw in an article two days ago that Symdeko would be shipped and in pharmacies as of yesterday. However, since this is a specialty drug, going through insurance and getting prior authorization from doctors will take some time.

  7. #7
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    I seem to recall with Orkambi, it was approved the beginning of July. I'd assumed we could just have our doctor send in a prescription and we'd be covered. Nope. We had to sign up with Vertex GPS because it wasn't even on BCBS' radar and Vertex only allowed 9 specific pharmacies to distribute the medication. Vertex GPS did the legwork for me getting forms to me and to our physician's office, communicated which pharmacy our insurance company chose for us and what the copay would be -- sometimes it's $0 other times it's $15. End of July the medication was shipped. So it took us about a month. IMO, contact Vertex GPS
    Parent to a child wcf double delta f508

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