Posted by Summer Katz, M.A., LMHC / Patient Advocate
The cystic fibrosis community has recently received some very exciting news! With the introduction of Trikafta, patients are beginning to see a breakthrough of tremendous potential with quick results. Trikafta is a combination of three drugs: elexacaftor, tezacaftor and ivacaftor, for patients (12 years and older) who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This accounts for 90% of cystic fibrosis patients.* The combination therapy essentially helps the protein made by the CFTR gene mutation function more effectively; showing an increase in quantity and function at the cellular surface, resulting in increased CFTR activity.
Trikafta is a “triple-combination therapy” consisting of three different modulators — tezacaftor/ivacaftor (which make up Symdeko) combined with elexacaftor. At the 2019 North American Cystic Fibrosis Conference in Nashville, TN, the highlight was certainly the FDA approval of this new triple combination therapy. The previously offered CFTR modulators include Orkambi, Symdeko, and Kalydeco. Individually, these drugs have been working to reduce the production and thickening of mucus in the body.
Trikafta has the potential to be significantly more effective than current modulators. The unique aspect of this new therapy is that it works by correcting the malfunctioning CFTR gene to restore normal activity. An added benefit is that pregnancy rates appear to be increasing for women taking Trikafta, as mucosal reduction occurs throughout the body and not solely inside the respiratory system. This trend was initially reported with the start of the individual CFTR modulator therapies.
Trikafta appears to be offering the latest hope for an exciting transition in the cystic fibrosis community. While there are the limitations* for use of this new therapy, there is also a renewed faith that we are headed in the right direction.
With the start of another new year, may we all go into 2020 with excitement, readiness, and willingness to take on this next chapter of fighting the CF battle with everything we have! Wishing each of you New Hope for a New Year!!
Let’s highlight our experiences together. Please feel free to send me a request to join the Cystic Fibrosis Connections Facebook group to keep the discussion going.
It is truly uplifting to see some of the many articles and social media posts from CF patients and families, expressing their joy over their amazing results. Here’s a few posts so that you can see for yourself…
Summer Katz, M.A., LMHC
Licensed Mental Health Counselor
Cystic Fibrosis Pharmacy Patient Advocate
*Disclaimer: This blog is provided for informational purposes only (including brief topic exploration or reflection) and should not be used as a substitute for professional mental health or medical treatment. **All listed resources have been identified for supplemental reading only, and the Cystic Fibrosis Pharmacy nor Summer Katz, M.A., LMHC is neither affiliated nor endorsing the aforementioned published material.