Friday, July 3, 2015

"Ain't No Mountain High Enough"

24 hours; I needed 24 hours to think about what the FDA's approval of Orkambi REALLY means. (read more on the approval HERE).
"Ain't no Mountain High Enough..."

Remember when Kalydeco was approved?  Approval of Kalydeco seemed like a no brainer since the GIANT improvements in lung function were across the board for CF patients. 

What is KALYDECO (ivacaftor)? (as seen on

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 2 years and older who have one of the following mutations in their CF gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.
KALYDECO is used for the treatment of CF in patients age 2 years and older who have an R117H mutation in their CF gene.
KALYDECO is not for use in people with CF due to other mutations in the CF gene. KALYDECO is not effective in patients with CF with two copies of the F508del mutation (F508del/F508del) in the CF gene.

Kalydeco will not benefit Elena because she has two copies of the F508del mutation (which by the way, she has an excellent Harry Potter/Cystic Fibrosis tattoo idea that goes along with her seen below!) 

Kalydeco was a major break thru for CF research because it proved that CF could be "put on hold" and even help to improve the CF patients overall health!  It is not a cure, but it is bridge that will buy us time to reach that cure.

So what is Orkambi and where do they come up with these drug names?

Orkambi is meant for CF patients that have two copies of the F508del mutation like Elena!! There are printed studies that show very small improvements in lung function.  The HUGE improvements we see in Kalydeco are not seen with Orkambi.  Yet, this is still a drug that seems to "put on hold" any further destruction of lungs.  Orkambi appears to be another bridge to give us the extra time we need to fight, fund raise, and raise awareness of Cystic Fibrosis. 

We are in the process of checking Elena's eligibility to get a prescription for Orkambi.  Another game changer drug that has been passed down through the pipeline.  The other drug studies behind Kalydeco and Orkambi are mind blowing and more exciting (even MORE exciting is what drug names they will chose next...maybe Bambikalydecambi?  See what I did there?)

"Ain't no mountain high enough" to keep us from fighting for the cure...even this potential price tag.
Thank you to all who have supported our family in so many ways since we discovered Elena's diagnosis.  This is a big event in CF history and I'm thrilled to have you all share in the celebration with us.  Cheers!

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