Genzyme hopes the FDA will approve their drug alglucosidase alfa (which is my ERT drug) on June 17,2010. The odds are mixed to whether or not this will finally happen as they have been seeking FDA approval for commercial use of this drug for years. They have experienced set-backs in the production of this drug at their manufacturing plant in Massachusetts.
Anticipating growing global demand, the company was moving production of Myozyme from Allston and a smaller facility in Framingham, Mass to a larger 4000 liter bioreactor scale in Geel, Belgium. However, upping production from 160 liter bioreactors to 2000L and 4000L scale-vats required that Genzyme submit supplemental Biologics License Application (BLA) to both FDA and European regulators, as the agencies insist that similar biologics produced in dissimilar bioreactors cannot be considered identical products, due to differences in the carbohydrate structures of the molecules in the so-called brews. Confused? That’s OK: the bigger tubs of alglucosidase alfa will continue to be sold under the Myozyme brand in Europe and under the Lumizyme title stateside. — BNET.Com, November 19, 2009, “FDA Delays Genzyme’s Lumizyme, Manufacturing Problems Blamed” *
What this means for Pompe patients is that, if it is approved those not currently receiving the drug through an ATAP study should be able to get it. And for those of us in an ATAP study, the study will end and we’ll join everyone else in the commercial use of the drug – and having to pay for it. Let the insurance battles begin!
To view the full story click here.
My friend Matt, who has been a constant source of information through his blog about his family’s journey with Pompe, has uncovered an interesting article from Forbes about Myozyme.
When people talk about expensive drugs, they usually are referring to drugs like Lipitor for high cholesterol ($1,500 a year), Zyprexa for schizophrenia ($7,000 a year) or Avastin for cancer ($50,000 a year). But none of these medications come close to making Forbes’ exclusive survey of the most expensive medicines on the planet.
The nine drugs on our list cost more than $200,000 a year for the average patient who takes them. Most of them treat rare genetic diseases that afflict fewer than 10,000 patients. For these diseases, there are few if any treatments. So biotech companies can charge pretty much whatever they want.
Myozyme, manufactured by Genzyme, makes Forbes’ list. Every two weeks I journey to the University of Florida to receive my infusion of Myozyme. Genzyme sends the tiny bottles of Myozyme to the pharmacy in powder form. The pharmacy will not start mixing the Myozyme for the IV bag until I contact them to tell them I am on my way. Mixing the Myozyme takes an hour and once the powder is mixed with the solution it is only stable for a number of hours. If the patient doesn’t show up, the very expensive drug has to be disposed of.
Myozyme, which inspired the Harrison Ford movie Extraordinary Measures*, costs up $100,000 for a child. But according to Genzyme, the average cost of adult treatment is $300,000 a year.
That is $300,000 a year for the rest of my life! Currently, my treatments are free as Genzyme has made the drug available to some adults through clinical trials as they await final FDA approval. Genzyme is optimistic the FDA will approve the drug for commercial use this summer which means I will have to start paying for my treatments, but it also means the drug will be available to adults with Pompe who don’t qualify for the clinical trial and therefore are not able to receive treatment at this time. FDA is scheduled to inspect Genzyme’s manufacturing plant in June, so we’ll soon know more about the fate of Myozyme and when it is going to start costing me $300,000 a year. Because of this price tag I will most likely live in poverty the rest of my life, but at least I will be able to live a life thanks to the magic medicine.
The price could come down as other pharmaceutical players join the game, but that is many years off and the price is not likely to plummet to Wal-Mart prices. Both Biomarin and Zystor are working on developing their own enzyme replacement therapy (ERT) drugs for Pompe, but they are both in the initial testing phases. I contacted Biomarin back in March 2010 to inquire about their progress and they responded with:
BioMarin Pharmaceutical Inc. has begun a program to develop an enzyme replacement therapy for Pompe Disease. Please note that this candidate molecule (BMN-103) is still in preclinical or animal testing. Further details of the product development timeline have not been announced and no further information is available at this time.
According to ZyStor’s website:
Despite the strong sales of these drugs [ERT], they are generally characterized by varying degrees of suboptimal ability to target the lysosome, need for the infusion of high doses and difficulty in manufacturing.
ZyStor’s lead product candidate, ZC-701, is an enzyme replacement therapy for Pompe disease that, based on pre-clinical evidence and manufacturing experience to date, overcomes these limitations.
In the future, more versions of ERT may be available and, according to what ZyStor seems to be working on, the newer versions of ERT may work even better than the current drug. If Genzyme, ZyStor, and Biomarin all have Pompe drugs on the market then I suspect they will continue to try and out-do one another by improving their respective drugs. Although all these possibilities are a long way off, they really do offer hope to the Pompe community.
You can read the entire Forbes’ article here: The World’s Most Expensive Drugs
*Extraordinary Measures will be available on DVD on May 18.
I had another Myozyme infusion yesterday, and as par for the course it went well again. I am very grateful for that. We are out of the “danger zone” as far as looking for an adverse reaction with the initiation of the infusions. That doesn’t mean I couldn’t still develop something, but the chances of that now are greatly reduced. Or at least in theory as technically speaking Myozyme is a clinical trial drug for adults. I had my own private room this time, which was nice. I could turn off the lights and snooze in the bed or watch TV. Which is no big deal since daytime television leaves something to be desired.
I am scheduled to start physical therapy next week. I’ll meet with a doctor at the facility and from there we’ll develop a regular schedule. Now comes the really hard part – the physical part. Being hooked up to an IV every two weeks is easy because the nurse and the IV pump do all the work. Physical therapy will probably wear me out – but in a good, healthy, productive way. Maybe I’ll even start to show small signs of improvement.
That being said, a lot of people have asked me if I’m feeling better after my infusions. Honestly, I can only say that I’m not feeling worse, but after a year of going downhill rapidly, not feeling worse for a few months is a
tremendous improvement. I have only had four infusions and it is still way too early to tell. The Myozyme, while it often referred to as the Magic Medicine, takes time to work. It will not “fix” me after just a few sessions. It could take months or even years before I see/feel an improvement and that is IF there is any improvement. This form of enzyme replacement therapy (ERT) is very new and as I mentioned is still technically in the clinical trial form for adults. The drug was only recently approved by the FDA for commercial use in children. Patients have not been on the drug long enough to really allow doctors to make any real predictions about how the drug will help and each case is very different. In essence all of us adults on ERT are lab rats and the results are pending. But every step now, is a step in the right direction – the long, long road of fighting Pompe. At least now with ERT I finally have a proper tool to fight with. Before it was like trying to fight a monster with a bubble wand – now I have a big club, so look out Pompe monster.
The Myozyme has a lot of work to do and it can’t do it alone. I have to help it through diet and exercise. My lack of the GAA enzyme has allowed glycogen to build up in my muscles for years, so the Myozyme has a lot of glycogen to pull out while keeping new glycogen from being stored at the same time. This is where the diet comes in. The less sugar and carbs I consume, the less glycogen needs to be stored and then fought by the Myozyme.
I think of my Pompe Disease as a runaway train. Until my diagnosis a few months ago, we had no idea how to stop it – or even what track it was on. The Myozyme helps slow the train down but it will never stop it completely. And the train has been running away for so long undetected that it will take time to slow down. During the train’s runaway days it caused some damage, some irreparable, along the way and we are now riding an ADA accessible transportation vehicle. Once the train has slowed to a reasonable speed the Myozyme can go back and try to repair some of the damage left behind. In the meantime, I can assist the Myozyme in its heroic efforts to stop the life threatening train by sticking to my diet and, soon to be implemented, exercise program. The train will continue to run away as no one knows where this Pompe Express will take us, but maybe just maybe it will slow down enough to let us relax a little enjoy the scenery. All aboard!
desserts? That is where I get creative. Sugar free desserts are not enough as those are usually still high in carbs. Breyers does make a low carb vanilla ice cream which has helped fight off those ice cream cravings. But berries or peaches with cream makes an even better low carb alternative to ice cream. And I love it!
- Cottage cheese
- Tuna or chicken salad
- String cheese
- Ham rolled with cream cheese
- Atkins bars
- Carrots and dip