December 21, 2007

Myozyme Improves Endurance, Pulmonary Function In Late-Onset Pompe

The Cambridge, Mass., biotechnology company Genzyme has determined that treating patients with late-onset Pompe disease (also known as acid maltase deficiency) with the synthetic enzyme Myozyme for 18 months increased endurance and pulmonary function.

The company announced these results Dec. 13.

The study involved 90 people with Pompe disease who were at least 8 years old at eight sites in the United States and Europe. Participants received either Myozyme, an enzyme designed to compensate for a deficiency of acid maltase (also known as acid alpha-glucosidase), or a placebo (inactive substance) by intravenous infusion.

MDA provided supplemental support for the trial.

After 18 months, participants treated with Myozyme increased the distance they could walk in six minutes by an average of about 30 meters (about 90 feet), while those in the placebo group showed no improvement from their baseline measurement.

A respiratory measurement known as forced vital capacity increased by 1 percent in the Myozyme-treated group, while it declined by approximately 3 percent in the placebo-treated patients.

The safety of Myozyme was found to be similar to that of an intravenously infused placebo.

Genzyme says it’s completing an analysis of this study and intends to present more detailed results at medical meetings and in a scientific journal.