As many of you know, Genzyme (the company that makes Hannah’s biweekly infusion of Cerezyme) had to shut down production of Cerezyme back in mid-June. At the time, it was determined that the children with neuronopathic Gaucher’s disease and other type 1 children under 18 years old (as well as those with type 1 in a life-threatening siutation) would continue to receive infusions. Unfortunately, this left thousands of patients without infusions for what we thought was going to be just a few months.
The situation seems to be more severe than originally thought, and it could be 6 months before there is enough production for all the Gaucher patients in the world. On a side note, this has led two other companies to be able to fast-track their Gaucher type 1 drugs through the FDA.
There is a “rumor,” and I stress again, it is JUST A RUMOR, that now there may not be enough drug for nGD and type 1 kids. Until I hear from my contact at Genzyme, I am not going to believe it. There have been so many rumors since this whole thing began that I just have to hear it “from the source.”
But what does scare me is the backlash and snowball effect from this Cerezyme shortage.
I had been working with the National Gaucher Foundation to see if we can start pushing type 2 and type 3 research. Things were going great for a few months, and the NGF was incredibly receptive and gung-ho with this idea. A plan was starting to really come together.
This Cerezyme shortage has changed everything.
The NGF is going to be unable to focus on this type 2 and type 3 research plan until, possibly, the end of the year. I completely understand why, as they are incredibly overloaded with not only thousands of patients who are up-in-arms about not receiving their treatment, dealing with media regarding this, but they are now thrown into the middle of all this fast-tracking of the new Gaucher type 1 drugs through the FDA. I don’t blame them at all — just trying to understand this craziness from a patient’s point-of-view is terribly confusing – I can’t imagine dealing with thousands of people like me looking for answers.
But where does this leave Hannah and the other neuronopathic Gaucher kids, the type 2 and type 3 kids? There is hardly anything out there for us, and what is out there is so scattered and hard-to-find. What we were coming up with could have made a difference in the organization and advancement of the research.
But now we just have to wait… wait for this Cerezyme shortage fiasco to come under control, which could be many months from now.
I just hope that we have the time to be able to wait.
This sucks. There’s just no other way to say it. It sucks for all those who have been going without. It sucks for those who may have to go without in the future. I am so sorry that this pushes everything you’ve been working toward to the back burner, Carrie. It is incredibly frustrating to you and is just not fair. Hugs.
Sorry for the whole situation and that you have to wait to find out.
I am sorry for your frustration with the NGF. Rejoice for the Cerezyme you have. It is a blessing. My daughters are without it and the emotional strain is unbearable! Perhaps the work that is going on with the other drugs will provide an answer for Hannah. You cannot control what is happening and it does nothing but sap your energy you need for your family and Hannah.You are doing an amazing job, but you cannot take on things you cannot control. I learned that the hard way when my girls were sick.
No Day But Today!!!!
Hello, my name is Anabela, I live in Portugal, I know that is not easy what is going on, we believe that soon everything will regresar normality with Cerezyme, for Gaucher’m sick and do therapy with Cerezyme to 9 years and now everything is uncertain, many doubts and few explanations! Strength to your daughter, and believe that everything will go well.