How I’m Preparing for Trikafta’s Expected Approval for Children Ages 6-11

I am anxiously awaiting the U.S. Food and Drug Administration's approval of Trikafta for children with cystic fibrosis ages 6 to 11. To prepare, I'm doing everything I can to ensure my 11-year-old son can take advantage of the medication as soon as it is approved.

| 4 min read
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Breck Gamel
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Results from a recent clinical trial provide reassurance that Trikafta® is safe for children ages 6-11. There is strong confidence within the CF community that the U.S. Food and Drug Administration (FDA) will soon approve Trikafta as the next highly effective modulator therapy for children with cystic fibrosis ages 6-11 with one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on lab data. This is very exciting to me as my son Bennett, 11, may benefit from this medication.

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I've been waiting for this decision for years. And, now that we're getting closer to the FDA's decision (expected by June 8), I have been feeling anxious about being ready.

Here are some of the things I've done to be prepared:

  • I have confirmed with my son's CF care team that his mutations make him eligible to have access to Trikafta, if approved by the FDA.
  • I scheduled Bennett's last CF clinic appointment as an in-person visit because the CF team prefers to see patients before prescribing new medications. (Since Trikafta will benefit a large number of pediatric CF patients, I wanted to make sure he could get an appointment.)
  • I asked his CF doctor to do liver blood tests required before starting Trikafta. High liver enzymes can be a concern for some on Trikafta, so these are typically checked before taking the medication. (We'll continue to check liver enzymes as Bennett is on Trikafta.)
  • I asked his CF care team about any drug interactions. As of now, Bennett doesn't take medications that would negatively interact with Trikafta use, but it's good to know that grapefruit juice is not recommended.
  • I requested a referral to an eye doctor from the CF care team to check for any abnormality of Bennett's eye lens in preparation for Trikafta treatment. Although most people do not have any trouble with cataracts, it is a possible side effect of modulators for pediatric patients. CF clinics monitor this symptom while children are on modulators.
  • I asked the CF care team how long we should prepare to wait to get Trikafta, once it's been approved. I learned it could take up to several weeks because of the logistics of having the medication ready to be dispensed and the need for insurer approval -- that is why I'm doing what I can in advance to avoid adding to the wait time.
  • I have started having ongoing conversations with my child about his feelings about Trikafta. I am trying to prepare him for possible changes to his body and to his treatment routine, such as taking the new Trikafta pills each day. I have also spoken to Bennett about possible side effects.
As excited as I am, I recognize that Trikafta doesn't work for all patients. I am working to make sure Bennett knows that he is not responsible for whatever happens to him on Trikafta and that he will not have failed his family if it doesn't work for him.
  • Lastly, I have focused on getting myself ready for my child to be on this new medication once it's available. I want to be realistic about my expectations. It might not work well for him. There may be fewer benefits of the medication for him than in other patients, for example. Or there may be intolerable side effects that require Bennett to stop the medication. And yet, it may be a medication that completely changes his life for the better. We won't know what will happen in Bennett's body until he is given a chance to try it.

Trikafta isn't a cure. The future of my son's health is still unknown. Only time will tell. But, I know one thing: We want to be ready for Trikafta as soon as it's available.

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Topics
CFTR Modulators | Parents & Guardians
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Breck is a mother of three children, including Bennett, her 11-year-old with cystic fibrosis. They live in Texas, along with their dog, Jellybean. Breck co-leads the Cystic Fibrosis Learning Network and previously chaired the Cystic Fibrosis Foundation's Patient and Family Research Advisory Council. Breck regularly blogs about her family's CF journey at initforbennett.com.

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This site contains general information about cystic fibrosis, as well as personal insight from the CF community. Opinions and experiences shared by members of our community, including but not limited to people with CF and their families, belong solely to the blog post author and do not represent those of the Cystic Fibrosis Foundation, unless explicitly stated. In addition, the site is not intended as a substitute for treatment advice from a medical professional. Consult your doctor before making any changes to your treatment.