When praising CF medications, Orkambi deserves a mention

Note: This column describes the author’s own experiences with Orkambi, Trikafta, and Symdeko. Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy.

In December 2019, I was working through my final year of a master’s degree in creative writing. Most of the time, I was focused on my thesis and teaching rhetoric and composition to undergraduates, a first in my career.

I worked at a corporate bookstore, where my favorite part of the job was talking to customers. We discussed books they already liked so that I could recommend new ones for them to discover. At the time, I had been taking the medication Orkambi (ivacaftor/lumacaftor), for my cystic fibrosis (CF), for four years.

Like many other kids with CF, I’d grown up always wishing for one thing: to be “normal” like my able-bodied friends. I’d see them go away to summer camp and know that my medical treatments made it harder for me to coordinate such an excursion. Plus, the risk of germ contamination from sharing a living space with other kids was too risky, as my mom often explained.

I avoided eating at buffets (and still do, because they’re gross) because I’d get sick when we did.

Perhaps most impactful of all, by the time the U.S. Food and Drug Administration approved Orkambi for patient use in 2015, when I was 20 years old, my baseline had declined so much that I had to be hospitalized at least twice a year. Orkambi was the first medicine I could access that gave me glimmers of hope for “normality” in my everyday life.

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A ‘bridge’ medicine

I started taking Orkambi when it first came out, and although we had hoped it would increase my FEV1 lung function (which stands for forced expiratory volume exhaled in the first second and is the primary clinical number most doctors focus on), as time stretched on, it became evident that this wouldn’t be my experience. My lung function continued to hover around the low- to mid-70% range. What it did do, though, was reduce the number of hospital admissions and increase my quality of life immeasurably.

Being able to stay out of the hospital for longer stretches of time gave me the stability to go to graduate school and the energy to engage in some semblance of a social life outside of academics.

When Trikafta (elexacaftor/tezacaftor/ivacaftor) came out in 2019, I was worried that changing medications would be problematic, as a previous attempt at switching to Symdeko (tezacaftor/ivacaftor) had been. In that case, I had what is colloquially known as “the purge,” in which the medicine works to undo the structural cause of our CF symptoms and forces us to cough up years of built-up gunk in our lungs.

After about a month of taking Symdeko, I would often cough so hard that I would vomit. I decided to switch back to Orkambi, which I continued to take until I switched to Trikafta in December 2019.

I am forever grateful for Orkambi, which I think of as a “bridge” medicine. Without it, my health undoubtedly would have continued to decline at a steady and rapid pace. Trikafta has been a big help, but I suspect the damage from CF would’ve been too great by the time it was approved had I not taken Orkambi.

There’s a lot of hype about how great Trikafta is, and in my opinion, the praise is warranted. But it also seems important to highlight the medications and other treatments that keep us CF patients going until our “miracle” drug arrives.

Note: Cystic Fibrosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Cystic Fibrosis News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to cystic fibrosis.