FDA won't sign off on Genzyme plan yet
Genzyme Corp., the maker of treatments for rare genetic disorders, said it failed to win approval for Lumizyme, a version of its drug for Pompe disease made in larger batches.
Genzyme must reach an agreement with the Food and Drug Administration for a post-approval study of the treatment, the Cambridge-based company said yesterday. The company also said it must respond to an FDA warning letter citing deficiencies in its manufacturing plant that would produce the larger batches of the drug.
Genzyme sells Myozyme for children and infants with Pompe disease, which leads to a buildup of sugar in the body that can harm the heart, liver, muscles, and nervous system. The company said it has asked regulators to approve Myozyme in 2,000-liter containers under the name Lumizyme for adults in addition to the 160 liters now allowed. Genzyme said the warning letter was "unexpected" because it had responded Oct. 31 with a plan to address regulators' requests and was on schedule to finish its corrective actions by March 31.
"We have made an enormous effort for more than two years to make this product broadly available in the United States, so we are obviously surprised and disappointed by this further delay," said Genzyme chairman and chief executive Henri Termeer. "We are confident we will be able to resolve all remaining issues with the FDA within three to six months."
Genzyme fell 6.2 percent to $53 at 5 p.m. in extended trading after a drop of $4.41, or 7.2 percent, to $56.52 in Nasdaq Composite trading. 