CF Community Reacts to Approval of Orkambi

Earlier this month, we learned that the new CF drug Orkambi was approved by the FDA. This was such an exciting, memorable and emotional day for everyone in the CF community. We heard from many who were thrilled about the potential for this drug to help people with CF and also heard from those who would not benefit from this breakthrough. Following are some reactions from those this impacts the most: people with CF and their loved ones.

Jul. 22, 2015 | 5 min read

Danielle Cipriani

Boom! Approved!!!! The gloves are off and the fight is on! Just Bring it CF! Meet your equalizer Orkambi!!! Congrats to all my delta f508 buddies
- Anna Payne | Facebook

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Right now, people with CF are skydiving without parachutes - once the disease hits its breaking point (and this happens at a different age for everybody), our plummets are fast and unavoidable. Orkambi is our parachute. Orkambi can slow us down. Orkambi can save us.
- Hannah Buck | hannahbreathes

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Where do I start?!? I'm sitting on a cruise in the middle of Alaska - no cell reception...no internet...yet still....somehow I get a message that the new cf drug that will slow the progression of this disease for Gavin and Jake has OFFICIALLY BEEN APPROVED!!! What?!? It's here?!? All of the blood, sweat and tears (not to mention massive amounts of cold hard cash) that so many of you have given to the Baker Boys' Battalion over the past 13 years is working! Seriously, guys....we're doing it.....we're saving lives. Orkambi....this new drug with a crazy ass name will give us more time....more days....more weeks....more months....more years....to find the CURE! It's really happening....it's really here....and I'm really sitting on this cruise in the Internet cafe looking like a crazy person with tears streaming down my face.....my heart is feeling very full of hope because this might be the very first time since 2002 that I have felt brave enough....strong enough.....safe enough to believe in a future - a real future beyond 40 years- for my boys. I'm not sure what to do with all of these emotions I have coursing through my body, but I do know that tonight I will raise a glass (maybe several) in sincere gratitude to each of you for allowing me to know what this feels like. <3 <3 <3 <3 <3
- Pam Baker | Facebook

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HUGE NEWS! This benefits 50% of CF patients with ONLY homozygous DF508. Since I am heterozygous DF508, this won't benefit me at this time. Happy but sad.
- Kristina de Bree | Facebook

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Today is amazing simply because it represents yet another victory of our incredible community over this deadly little monster called cystic fibrosis. This approval, in other words, represents the strength we have in numbers (however small we might seem) and the breathtaking tenacity, courage, and commitment of our team against what sometimes can feel like an unstoppable enemy. It represents US, beautiful people. Because believe it or not, WE made this happen.
- Piper Beatty | A Matter of Life and Breath

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Tears for my angel that never was able to receive these amazing drugs…and tears of JOY for my son <3
- Margarete Cassalina | Facebook

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Wonderful day in the CF community, however, it comes with mixed emotions. From an insurance/Medicaid standpoint, I know we have our work cut out for us.
- Marissa Benchea | Facebook

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I had the privilege of participating in both the phase 2 and phase 3 trials for the combination of Kalydeco and Orkambi. Ultimately, I had to drop out of the phase 3 study because Orkambi wasn't the right fit for me. I am still celebrating today for all those with CF who will be helped by this drug. I know my drug is out there because I know the CF Foundation will not rest until there is a cure for all people with CF.
- KC White | email to the Foundation

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Great news today in the treatment of Cystic Fibrosis! It is one of the biggest medical advancements I have seen in my lifetime for the daily fight against CF that I live with along with my brother.

This medicine has been found to have drastic improvements on quality of life for those that have been participating in the study for it over the past several years. I have prayed so incredibly hard for this medicine to hit the market while we are still healthy enough to benefit from it.

While it is not guaranteed that this will replace lost lung function, it is an incredibly hopeful reality that it will drastically slow any further decline.

While this is a great success for the largest portion of the CF population, there are so many who are close to me with other, more rare, mutations, and I hope and pray that the continued development of technologies will reach EVERY person with CF #‎SoonerThanLater!

Thank you #‎Vertex and #‎CFF!

- Brian Callanan | Facebook

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.

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Topics

Research | CFTR Modulators | Drug Pipeline

Danielle listens to and partners with people with CF and their families to create meaningful opportunities for connection. She also leads the creation of online spaces and events for people in the CF community to connect, learn, share and inspire each other. Danielle earned her B.A. in communications from American University in Washington, D.C. She currently lives in Littleton, Colo., with her husband Vince and dog Baci.

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