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The currently approved Myozyme is manufactured by [[Genzyme|Genzyme Corp.]] in Cambridge, Massachusetts, USA. Its devlopment was a complex process. Genzyme first partnered with [[Pharming Group NV]] who had managed to produce acid alpha-glucosidase from the milk of transgentic rabbits. They also partnered with a second group based at [[Duke University]] using Chinese hamster ovary cells. In 2001 it acquired Novazyme which was also working on this enzyme. Genzyme also had its own product (Myozyme) under development. In November 2001 Genzyme chief executive Henri Termeer organised a systematic comparison of the various potential drugs. It was found that Myozyme was the most efficatious and easiest to manufacture. Work on the other products was then discontinued.
The FDA has approved Myozyme for administration by intravenous infusion of solution into a vein. The safety and efficacy of Myozyme were assessed in two separate clinical trials in 39 infantile-onset patients with Pompe disease ranging in age from 1 month to 3.5 years at the time of the first infusion. Myozyme costs an average of $300,000 a year, and must be taken for the patients' entire life. The treatment is not without side effects which include fever, cardiac failure, pneumonia, respiratory failure and rarely shock. Some insurers have refused to pay for it.<ref>[http://online.wsj.com/article/SB119007210553130427.html Burden of Proof: As Costs Rise, New Medicines Face Pushback; Insurers Limit Coverage To FDA-Approved Uses; $300,000 Drug Denied] By Geeta Anand, ''[[The Wall Street Journal]]'', September 18, 2007.</ref> The treatment is better at stabilizing the clinical condition than reversing the pathological changes that have already occured. Early diagnosis leads to better outcomes.
On August 14, 2006, Health Canada approved Myozyme for the treatment of Pompe disease. On June 14, 2007 the Canadian Common Drug Review issued their recommendations regarding public funding for Myozyme therapy. Their recommendation was to provide funding to treat a very small subset of Pompe patients (Infants less one year of age with Cardiomyopathy).<ref>[http://www.cadth.ca/media/cdr/complete/cdr_complete_Myozyme_June-14-2007_e.pdf] Canadian Common Drug Review Recommendations on Public Funding for Myozyme </ref> The vast majority of developed countries are providing access to therapy for all diagnosed Pompe patients.<ref>[http://www.lexdon.com/article/Genzyme_Reports_Financial_Results_for/45177.html] Genzyme received broad approval in the European Union</ref>
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