For those of you in my Pompe family this is old news, but I haven’t had a chance to update my non-Pompe readers yet. The FDA granted Genzyme approval for Lumizyme, the adult enzyme replacement therapy for Pompe Disease. The approval came a few weeks ago, but I’ve been too busy to catch everyone up.
What this means for me is that my insurance company will now have to approve the treatment and will receive a bill for my treatments – a very, very large bill. The University of Florida and Genzyme are currently negotiating approval with my insurance company so that I may continue my treatments. What this means for patients not currently being treated for through the ATAP program is that they will soon be able to start their enzyme replacement therapy! For those of you in that boat – congratulations! The treatments have already started to improve my condition and I hope you all can say the same thing too soon!
I will continue to receive my treatments at the University of Florida until another infusion site can be set up. From what I understand, they are focusing on setting up the first Florida site in the Orlando area. Doctors, nurses, and pharmacists all have to be trained to administer the newly approved drug. Many have never heard of it, let alone know how to prescribe, mix, or administer it. The hospital has to agree to take on the responsibility of administering this very expensive drug that has no real long term study data. And the infusions have to take place in a hospital setting in case someone has a severe reaction. Can you say somewhat risky from a pencil pusher hospital admin point of view? So it can’t just be set up anywhere nor everywhere. With only a handful of confirmed Pompe patients (at last count there are 10-12) in the state of Florida, one site in the middle of the state does make sense. The FDA will continue to monitor us, the doctors and Genzyme are required to continue to provide patient information to the FDA. So in essence the “study” will continue, it is just that the drug is now commercially available to all who are fighting Pompe.
Last week I officially registered with the Orlando area Muscular Dystrophy Association and met with their clinic doctor. He has met with Genzyme and will be going through the training to administer Lumizyme. I have no idea how long the training will take or when I will have to make the transition from the University of Florida to the Orlando site, but for now I have infusion appointments scheduled at the University of Florida through August.