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Manufacturing woes plague launch of Myozyme
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Genzyme has watched its share price slide recently as it struggles to gain FDA approval for a new manufacturing facility to make Myozyme, a super-expensive enzyme-replacement therapy for Pompe disease. Genzyme says that it's urging patients to get doses through the unapproved facility that produces the therapy for experimental uses. But that source is free, instead of the $300,000-plus a year that the drug costs on the open market. The Wall Street Journal cites a knowledgeable source who says that Genzyme's problem is its inability to produce the therapy in the same version made for clinical trials. Genzyme plans to resubmit its application later this year--provided it can make matching therapies. About 10,000 people in the world have Pompe disease, and about 700 are taking Myozyme.
- read the report from The Wall Street Journal (sub. req.)
Related Article:
Genzyme wins Myozyme OK. Report
Myozyme approval leads to rich royalty pact. Report
Biotech drug prices hitting ceiling? Report
Comments
By Bo Piela | Posted 3:41pm | August 15, 2007
Genzyme provided some clarification on this topic in our letter published today in the Wall Street Journal. Wanted to be sure everyone saw this.
By yousef | Posted 4:26am | June 24, 2009
In the name of God
To whom it may concern
I need your help
First of all, thanks for any efforts that you made for me!
Greetings!
Hello! I hope you, the respected physicians, to have a good time
I am Mr. Yousef Vazifeh, 25 years old (born on 10/05/1984), from Tehran (Iran).
After some medical tests which were carried out on me because of myopathy (progressive muscle weakness), and transferring my blood sample to Germany, it was revealed that I am suffering from a genetic illness called Pompe’s Disease that is emerged due to shortage of some enzymes like Enzyme Acid-alpha- glocosidase (EC 3.2.1.20) in the body.
According to Iranian physicians, as if the only cure for the given disease is to inject intravenously Myozyme (Alglucosidase alfa, rhGAA) ampoule whereas this medicine does not exist in Iran and it is exclusively produced by some foreign leading companies as well as the exorbitant price of the mentioned drug (based on Iranian physicians’ comment, it is about 200-210 thousands dollars per year), thus I am not afford to pay such a cost. I hereby ask you dear sirs to read my medical documents which I send them for you and after your professional confirmation, I request you to guide and help me in providing this medicine with better conditions and lower cost to remedy such congenital disease.
Furthermore I should tell the fact that my older brother was died due to mis- prognosis such disease or it might be because of no avail to this drug, in 2003.
Additionally, I am ready for any kind of cooperation and/ or doing surgical operation for my remedy.
I need your helps!
Thanks!
Sincerely Yours,
Yousef Vazifeh
Regards!
Furthermore, because of the fact that I rarely know English language so that firstly I have such letters to be translated and then I could send your reply; thus for this reason it may be late to answer your letter
Full Name: Mr. Yousef Vazifeh
Gender: Male
Weight: 52Kg
• You can contact me by the following addresses, if required:
Email: Yousef_teh@yahoo.com
and/ or panda12347@yahoo.com
Address: No. 32, beside Daevoo repair center, Northern Takestan St, Satar Khan St, Tehran, Iran. Postal Code: 1444763541
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