Wall Street Journal Examines High Price of Gaucher Disease Treatment Cerezyme

The Wall Street Journal on Wednesday examined the high price of Cerezyme -- a treatment for Gaucher disease manufactured by Genzyme -- and profiled a patient with the disease. According to the Journal, FDA in 1991 approved Cerezyme as an "orphan" medication after Genzyme licensed the rights to the missing enzyme in Gaucher disease from NIH and conducted positive clinical trials. Under the Orphan Drug Act, passed in 1983 to encourage the development of treatments for rare diseases, pharmaceutical and biotechnology companies can market medications with orphan status exclusively for seven years; the law also provides companies with tax and fee incentives and access to federal grants. FDA awards orphan status to treatments for diseases that affect less than 200,000 U.S. residents annually. Cerezyme, which costs about $200,000 annually for the average patient, had $840 million is sales in 2004, with a more than 90% gross profit margin (Anand [1], Wall Street Journal, 11/16).

Reasons
The Journal on Wednesday also examined the reasons for the high price of Cerezyme. According to Genzyme officials, the high price of Cerezyme allows the company to continue to develop other medications and fund programs that provide small amounts of treatments at no cost. In addition, Genzyme CEO Henri Termeer said Cerezyme was difficult and expensive to manufacture when the medications first reached the market. At the time, Genzyme used 22,000 human placentas to manufacture one annual treatment of Cerezyme, and the medication cost the company $1.90 for every dose sold for $3.50 (Anand [2], Wall Street Journal, 11/16).