Pompe disease is a rare inherited enzyme deficiency that destroys muscles and can kill infants at a very young age.
Pompe disease is a rare inherited enzyme deficiency that destroys muscles and can kill infants at a very young age. Myozyme is a drug developed by Genzyme to treat this disease. Genzyme, a biotechnology company in Cambridge, Mass said that it has spent more than $500 million since 1998 to develop Myozyme.
It has also received incentives provided by the federal government to develop medicines against such disease collectively called orphans disease. The drug after passing through a variety of tests and trails has won the federal approval to sell the drug in the market. Genzyme officials said that the dug Myozyme, would cost about $200,000 to $300,000 a year. The disease incidence is less than 10000 people and they are all grateful for Genzyme's drugs. Genzyme said that it will provide the drug free of cost to those who cannot pay for it. During the clinical trail from 2003-05 the drug was administered to 18 infants.They all lived until they were at least 18 months old, or about a year after starting treatment, and most are still alive today. The warning on the drug label says that Myozyme can also cause life-threatening anaphylactic or allergic reactions. Dr. David Meeker, president of the Genzyme division that developed Myozyme said that a new blood test was offered to diagnose the disease rapidly which could lead to earlier treatment with better outcome. But further tests are necessary to determine the effect of the drug against Pompe disease that begins later in childhood or in adulthood.
