The US Food and Drug Administration has approved a biologics license application (BLA) for Myozyme (alglucosidase alfa, rhGAA), the first treatment for patients with Pompe disease, a rare but severely debilitating disease.

Pompe disease, which affects one in 40,000-300,000 individuals, drastically reduces a person's muscle and respiratory function. Myozyme, manufactured by Genzyme Corp., had been granted FDA Orphan Drug designation and was approved under a priority review. Orphan products are developed to treat rare diseases or conditions that affect fewer than 200,000 people in the US. The Orphan Drug Act provides a seven-year period of exclusive marketing to the first sponsor who obtains marketing approval for a designated orphan drug.

"This approval is another example of the benefits of the Orphan Drug program, which provides incentives for the development of treatments for diseases affecting fewer than 200,000 patients in the United States a year," said Dr. Steven Galson, the director of FDA's Center for Drug Evaluation and Research. "Until now, Pompe disease has had no approved treatment," he added.

Pompe disease is an inherited disease caused by the deficiency or lack of the enzyme acid alpha-glucosidase, which is essential for normal muscle development and function. The disease, which usually results in death from respiratory failure, is rapidly fatal in newborn babies.