Beautiful Mess

And BOOM!  It happened today! The FDA approved Kalydeco (VX-770) for use in patients with the G551D mutation of cystic fibrosis, who are at least 6 years old!!  The approval today is almost 3 months ahead of their April 18^th target review date.  This morning started out like any other--give Brady Prevacid and start treatments.  Treatments finished, we had moved onto breakfast plus a handful of pills.  He was eating his eggs and waffles when I saw the post on Facebook that the FDA had approved Kalydeco.   I read it and re-read it, and re-read it before I started making weird noises.  I lose my ability to form words when I’m that excited.  I could hear that Brock was on the phone upstairs in his “office.”  He is working from our rental house in Arizona right now.  I ran to the top of the stairs and started making more sounds.  He hung up the phone a few seconds later.  “What’s wrong?!!  WHAT IS WRONG?!” In his voice, I could tell he was scared Brady was hurt or something, but I was still unable to talk.   I ran back downstairs and hoped he would follow me.  Finally I spit out: “READ IT BROCK!  WHAT DOES THAT SAY?  DOES THAT SAY WHAT I THINK IT DOES?”  My heart was beating out of my chest.  My whole body, especially my hands, were shaking uncontrollably as we realized what was happening (my hands are still shaking now).  The next hour or so was filled with messaging on facebook, calling our families, and having wild emotional breakdowns.  “Mommy is sad.”  “No, Mommy is happy!”  It has been a confusing morning for Brady.  After the initial freak out, I realized I needed to take care of some business.  I made phone calls to Brady’s clinic, Vertex Pharmaceuticals, and my attorney.  I emailed the CEO of the CFF and listened to the Investor Conference call held by Vertex today.

See, this news is like a dream come true for us, but there is one little detail that poses a problem.   Brady is only 4 ½ years old and the drug is approved for children age 6 and up.  I learned that Kalydeco is going to have a $294K annual price tag per patient.  Since clinical trials have only been performed in kids age 6+, the insurance company would consider it “off-label” use and will almost certainly reject the claim.  We understand that there is a clinical trial planned for “mid-year 2012” for children aged 2-5 with G551D OR OTHER GATING MUTATION!  They wouldn’t spill any further detail on the specifics of that trial…which is going to give me a nervous breakdown.  Another source of mine believes the trial will begin dosing phases in 1-2 mo.  And trial phases a few months after that.  The trial is scheduled for 6 month duration and is a double blind placebo control.  Approval for this age group is estimated for early 2013.  This news hit me like a gut punch.  Because Brady is only 4 ½, we have a couple of options. 

1) Wait for the trial to begin.  Hope he can be enrolled in it and be in the 50% that receives the actual drug and not the placebo OR

2) attempt to get the drug “off-label,” which will entail a legal battle with the insurance company, but may potentially lead to Brady getting the real drug sooner.  After a few short hours of thinking on this I decide that I really only have one option.  There isn’t a chance in hell that I’m going to be able to sit around and wait.  If Brady hasn’t gotten the drug in the meantime, we will attempt to enroll him in the clinical trial.  But right now we’re going to do our best to get it for him and FIGHT FIGHT FIGHT!  Brady’s CF specialist is ready and prepared for the appeal process with the insurance company.  We are working on getting weight information on some of the 6-11 yr. old trial participants, so we can prove that a 150 mg. dose is appropriate for Brady (he weighs almost 50 lbs, which I’m sure is similar to some of the trial participants who were 6 years old.)  I have a huge knot in my stomach.  My hands are still super sweaty.  The idea of having a legal battle over a drug this important and this expensive makes me want to puke.  I know I’ve said this before, but I think if the drug is out there and others can start taking it and halting the progression of their disease and I’m told to sit around and wait for 6 months or a year for Brady to get it…then my head really might explode.  Vertex has scheduled another conference call this Thursday, where they said they will outline their plans for 2012, so I hope they bring more details to the table. 

Typically, I try to include some useful information in this blog, so I wanted to share some of, what I considered, to be the highlights from the Vertex Conference call I listened to earlier today. 

-Price is set at $294K annually per patient

-Vertex has a patient assistance program up and running: http://www.vertexgps.com

-They estimate that there are 1200 patients nationwide with G551D, 200 of those are under 6.  Expanding to other gating mutations would double that population.    

-The trial for 2-5 yr. olds will include all gating mutations and is scheduled for “mid-year.”

-They expect a positive European ruling in the next few months.  They applied for all gating mutations.  They aren’t sure if it will be approved for all gating, or simply for G551D, as in the U.S. 

-Price will be re-evaluated if Kalydeco becomes a part of the treatment regimen for all CFers in the future.

Please understand that I’m so tuned in to certain pieces of information, so I’m sure I’m missing some big points.  Feel free to listen for yourself at http://investors.vrtx.com/events.cfm

I’m going to write more when I can think straight, and when I get more info...  I really wanted to do a little writing today to commemorate what a mess I am.  How crazy it feels to receive a miracle.  I want to be clear about one thing.  How to get the medicine to Brady is such a good problem to have.  Kalydeco exists and is now being marketed for sale!  Jesus.  That is wonderful news!  I don’t want any portion of this blog to sound like a complaint, because I’ve never felt more fortunate.  I swear I’ve been floating somewhere above my body all day, looking down and watching this beautiful mess unfold.   What lies ahead is so scary.  We don’t have any idea what is going to happen next or how we are going to make sure Brady gets this medicine that he needs.  One thing is for sure.  If a big fight has to go down…I volunteer to get in the ring.  Seriously, put me in coach.  I don’t mind getting bruised up.  Those who know me personally realize I actually get a kick out of a good fight and backing down is not a part of my vocabulary.  And for my sweet Brady…well I think I’d do just about anything.  As a fighter, I know that I’m going to need a solid team in my corner to have a shot at winning.  I’m going to watch Rocky now for inspirado.  I hope to see you all back in my corner real soon.  I’ll end with a toast (since I’m already drinking)—Cheers to ANYTHING BEING POSSIBLE in 2012!