Fabry patients in need of Genzyme's Fabrazyme are challenging again. Last month, the challengers lost their attempt to get the NIH to exercise so-called march-in rights to allow another manufacturer to enter the market to make Fabrazyme - for which Genzyme hoolds the license to the patents. This request was denied due to the NIH's feeling that no alternative manufacturer could start now and bring drug to market before Genzyme could get back into fuller production.
Now two of these patients are asking the FDA to regulate how Genzyme allots its drug. It is asking the agency to make sure drug goes to the US market first before going to Europe -- apparently 62% of the drug is shipped overseas. The EMEA has licensed an alternative drug, Replagal, made by Shire for the treatment of Fabry's. The same drug is not licesned in the US and Shire as yet has not applied for one.
The US market is therefore short of Fabrazyme - where current patients subsist on a suboptimal dose in many cases and new patients can't get any drug - and has no access to Replagal. There have been 3 reported deaths of Fabry's disease patients in the US due to the shortage. No such deaths have been reported in Europe. Now the FDA is beginning to import Replagal to replace the Fabrazyme which was exported -- but patients must show "critical need" on a case by case basis to receive the US-unapproved drug.
Posted by Bruce Lehr Jan 20th 2011.