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Thread: Kalydeco with an FEV1 of ~27% (homozygous delta-F508)

  1. #1
    Bosh
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    Kalydeco with an FEV1 of ~27% (homozygous delta-F508)

    Treatment with Kalydeco on patients with the homozygous delta-F508 mutation will apparently pass FDA muster in July. My girlfriend is twenty-five and her doctor has indicated that her current baseline FEV1 is around twenty-seven percent, that this treatment is intended for those with an FEV1 of thirty-five percent or higher.

    Can someone elucidate what he's talking about here? I fully intend to fight tooth and nail for this treatment on its approval, but I'm scared there's something I don't know that will make this impossible.

  2. #2
    rb1973
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    Quote Originally Posted by Bosh View Post
    Treatment with Kalydeco on patients with the homozygous delta-F508 mutation will apparently pass FDA muster in July. My girlfriend is twenty-five and her doctor has indicated that her current baseline FEV1 is around twenty-seven percent, that this treatment is intended for those with an FEV1 of thirty-five percent or higher.

    Can someone elucidate what he's talking about here? I fully intend to fight tooth and nail for this treatment on its approval, but I'm scared there's something I don't know that will make this impossible.
    Take as deep of a breath in as you can, hold it for a second then blow it out as fast and hard as you can. The amount of air that comes out in the first second is your FEV1 - Forced Expiratory Volume in 1 second. A formula using your age, sex, height and weight, produces a predicted number of what that volume of air should be. Based on that, your girlfriend is forcing out 27% of what is predicted (basically what a person without any lung impairment should be able to blow). The Doc is saying individuals blowing greater than or equal to 35% would be eligible for the drug. If what he is saying is factual (I'm not sure what the fev1 requirements are), the problem is that the cost of the drug is expected to be in the ballpark of $330,000 a year per patient. Insurance companies will fight tooth and nail not to pay that and will use those FEV1 number requirements to their advantage to deny it. Basically if your below 35%, the drug either has not been tested on those patients or is ineffective. If I were in her shoes, I'd want to try it regardless. The problem is getting it paid for.

  3. #3
    jamest
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    Save your energy. Orkambi - the lamacaftor + ivacaftor combo intended to treat DDF508 - goes in front of the FDA for approval July 5th. No need to push for kalydeco at that point, there will be a ddf508 specific treatment available soon(ish) (hopefully).

  4. #4
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    I'm not sure exactly what the Orkambi labeling will include. I just know past studies only included those patients with FEV1 of 40% or higher.
    There is a Vertex study currently enrolling for ddf508 with FEV1 under 40%:
    https://clinicaltrials.gov/ct2/show/...vx+809&rank=11
    Hopefully there is a site in your area. If not, you may want to call vertex and ask about travel expenses covered.
    If study doesn't work out for you and your doctor is willing to write a prescription for Orkambi, than give it a try putting it through your insurance. There are some insurance companies that cover it even though you are considered "off label". You don't know until you try! If your doctor isn't willing to write a prescription off label, You may want to consider finding a doctor that will. As I'm sure you know, her lung function is very compromised and I truly empathize with the sense of urgency you feel. Although I don't share her mutations, my lung function went from 31% to 42% thanks to Kalydeco ( off label- wasn't expected to be effective for my mutation r334w and ddf508.)
    Study results have shown that Orkambi has shown a "modest" improvement in lung function. With serious lung disease, this "modest" improvement will likely translate to quality of life improvement that will hopefully be felt and appreciated on a daily basis.
    If you decide to try to put it through insurance and it is denied, I can help you with the appeal process if it comes to that.
    Keep up the persistence! It sounds like you're a great advocate!

  5. #5
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    Swamped at work, so sorry this is short.
    I echo Jricci's great advise. I'd just add that Kalydeco while noting the study was 40% and above did not limit its "usage" to those 40% or above (while it did expressly say not effective for ddf508). http://www.accessdata.fda.gov/drugsa...188s017lbl.pdf
    That's the labeling info.
    So I would hope her FEV won't effect prescription or insurance.

    Vertex also I think has a "compassionate use" program going for Orcambi which might get her access now. She should call her doctor and ask him/her to call Vertex re compassionate use. (I don't think a patient can, but she could try first and then tell doctor it has to come from him.)

    Finally, the study jricci noted above is "open-label" which means they are giving the drug to all participants, not a placebo...so that help convince a doctor it is okay to give it to her, if the doctor is hesitant.

    Good luck!

  6. #6
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    Quote Originally Posted by jricci View Post
    I'm not sure exactly what the Orkambi labeling will include. I just know past studies only included those patients with FEV1 of 40% or higher.
    There is a Vertex study currently enrolling for ddf508 with FEV1 under 40%:
    https://clinicaltrials.gov/ct2/show/...vx+809&rank=11
    Hopefully there is a site in your area. If not, you may want to call vertex and ask about travel expenses covered.
    If study doesn't work out for you and your doctor is willing to write a prescription for Orkambi, than give it a try putting it through your insurance. There are some insurance companies that cover it even though you are considered "off label". You don't know until you try! If your doctor isn't willing to write a prescription off label, You may want to consider finding a doctor that will. As I'm sure you know, her lung function is very compromised and I truly empathize with the sense of urgency you feel. Although I don't share her mutations, my lung function went from 31% to 42% thanks to Kalydeco ( off label- wasn't expected to be effective for my mutation r334w and ddf508.)
    Study results have shown that Orkambi has shown a "modest" improvement in lung function. With serious lung disease, this "modest" improvement will likely translate to quality of life improvement that will hopefully be felt and appreciated on a daily basis.
    If you decide to try to put it through insurance and it is denied, I can help you with the appeal process if it comes to that.
    Keep up the persistence! It sounds like you're a great advocate!
    I wrote this last post at 1:30 in the morning during a bout with insomnia, so I guess my thinking wasnít clear (thatís what Iíll blame it on anyway!) I agree with aboveallislove, FEV1 shouldnít affect labeling for Orcambi. In prior Vertex studies for Kalydeco, even though there were restrictions for FEV1 in order to participate in the study; this never affected the labeling once it was approved. When it is approved in July, I donít see a reason why your girlfriend wouldnít have access to it. Her FEV1 shouldnít affect this.
    But if you donít want to wait until July, then look into the study I mentioned or call Vertex to see if they offer a compassionate use program as aboveallislove suggested. Good luck to you.

  7. #7
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    I actually thought the labeling was limited..maybe it was in Australia or they were going to there? I had to pull to check.

  8. #8
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    My daughter just had her checkup, currently bounces from 29 to 33% fev1. She asked her doctor if she can start that when it's available and he said yes, that it would take a lot of authorizations on their part, but that it would benefit her. I specifically asked him, if the FEV1 had to be a certain number and he said no. That she qualifies. He's the director of our CF Center, and he's never been wrong with anything else so far. I hope that everyone can get it ASAP and not have to jump through hoops to get it approved.

  9. #9
    good luck ..happy to hear that

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