Myozyme: One of the most expensive drugs on the planet

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.

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