farbrazymeThe big buzz in the Rare Disease community over the past few days has been Genzyme’s announcement to halt production and shipments of Cerezyme and Fabrazyme, two drugs for rare genetic disorders Gaucher disease and Fabry disease, after a virus (Vesivirus 2117) was discovered in production equipment at its Boston area plant.  Here is the press release about this most unfortunate situation facing Genzyme and Gaucher and Fabry patients.
According to the Boston Globe, the drugs are used by 8,000 people worldwide and cost about $200,000 per patient annually, which make them some of the most expensive drugs in the world.  While the virus that hit the plant has the ability to taint the drugs, it is not considered harmful to humans.  As a result of the plant shutdown, the current supply of drugs for Gaucher and Fabry patients will need to be rationed and patients will need to be prepared to miss intravenous infusions, according to Genzyme.
According to a report by reporter Shirley Wang at the Wall Street Journal, Genzyme will lose between $100-$300 million dollars as a result.  This will directly result in less research and development at Genzyme which will hurt rare disease research overall.  This makes me sad. I like Genzyme and we already have limited money going into rare disease research efforts.
Many people in the rare disease community feel badly for Genzyme.  People are obviously angry about the situation — it’s more about “the bug” and less about Genzyme.  Without Genzyme stepping up to the plate and developing drugs for rare diseases like Gaucher and Fabry, many of these patients would not have treatments today.
According to the Globe and FDA spokesperson Christoper C. Kelly, last fall, an FDA inspection of the Allston plant found “significant deviations from current good manufacturing practice in the manufacture of licensed therapeutic drug products, bulk drug substances, and drug components.”  The CEO of Genzyme, Henri Termeer, was sent a “warning letter” in February.   It’s unclear if it was about this problem.  Unless it turns out that Genzyme was outright negligent, I believe the Rare Disease community will work to support this amazing company, help them get through this nightmare and forgive them.