When you live with illness your entire life, you get used to certain realities. With Cystic Fibrosis, the reality is this disease kills you. Thanks to research and medications funded by the Cystic Fibrosis Foundation, the life expectancy has risen substantially in my lifetime, but its still hovers at about 40 years old. Another reality is we don’t often go gently into that good night. Whether it’s respiratory failure, transplant complications, heart failure, or liver failure, end stages of Cystic Fibrosis are hardly forgiving.
Yesterday, my reality maybe changed. At the very least, it got put on hold. Around lunch time, my speciality pharmacy called to say my health insurance approved a prior authorization for the newly FDA-approved CF drug, Orkambi. In other words, my insurance agreed to foot the whopping $300,000 annual bill for the prescription. I nearly fainted at the news.
If you’re not part of the CF community, maybe you haven’t heard of Orkambi. I’m here to tell you, it’s a big deal. When I was born in 1988, they didn’t even know what or where the CF gene was. Now, 27 years later, Orkambi is the second drug to get at the underlying cause of CF. The first drug to treat the underlying cause was Kalydeco. While a breakthrough in CF research, Kalydeco was only successful for a rare mutation affecting about 4% of CF patients. Though Orkambi is not an all-encompassing drug (it does not work for all CF mutations), it does target the mutation found in almost half the CF population. Rather than just treating symptoms of the disease, Orkambi helps the defective CFTR protein function normally for those with two copies of the F508del mutation.
During drug trials, researchers noted a 40% decrease in lung exacerbations. It’s those tricky lung exacerbations that lead to inflammation and infections, decline in lung function, damage to the lungs, and ultimately, death for Cystic Fibrosis patients. A 40% decrease is life changing.
I’ll be starting Okrambi in the next two weeks and as I type this I can’t believe it’s real.
Orkambi is not a cure, regular daily treatments and clinic checkups are still required, and as mentioned, it is not effective for all mutations of CF. But cure or not, it feels like I’m getting a new lease on life. Having kids and actually sticking around to raise them feels less like a daydream. Growing older doesn’t sound as scary because maybe I won’t get sicker with each passing year. Maybe I’ll still be here when they cure this thing.
My life leading up to Orkambi has hardly been all doom and gloom. I have an incredible support base of family and friends. A beautiful and loving boyfriend and an even more beautiful and loving golden retriever. I’ve traveled, been on a few adventures. And on the health front, I’m doing well. My lung functions are up and I can run a full three miles without wheezing. All in all, I really, really like my life. But it’s hard to shake a weight as heavy as chronic illness. Like many people with CF, I struggle with anxiety and at times, depression. Some of my anxiety probably stems from my fear of infection, from my fear of getting worse. Every flu season I hold my breath and hope I can evade it. Every flight I flinch at any passenger sniffle. I worry so much about getting sick, sometimes I actually make myself sick with all the worrying.
With Orkambi and its possible 40% decrease in lung exacerbations, for the first time in a long time, I feel light, unburdened. I feel hopeful.
I’m not the only one feeling hopeful. Yesterday, when I called my parents with my Orkambi news I could practically hear their hearts bursting over the phone – and from 2000 miles away. The fight’s not over. CF is still scary, it’s still a killer, and I still have it. But I think my family is due at least one long sigh of relief.
Before I start planning my bright(er) future, there might be some side effects. Maybe even brutal ones. Some CFers call the first week on Orkambi “hell week” or “the purge.” Excessive cough accompanied by boatloads of mucus, bloody sputum, and at times, vomit, are all common experiences in the first weeks on this pill. And while I’m not particularly looking forward to any of that, I can’t shake this feeling of lightness.
Plus, I’ve been feeling crappy for going on 28 years – what’s a few weeks?