Pompe's disease

OKLAHOMA CITY (JR) -- Novazyme Pharmaceuticals on Thursday said that its phosphorylated enzyme replacement therapy for the treatment of Pompe Disease has been granted orphan drug status.

The Oklahoma City-based company received formal notification from the Office of Orphan Products Development at the Food and Drug Administration.

This notification from the FDA represents the first official validation of Novazyme's core phosphorylation technology platform to address the needs of lysosomal storage disease patients, said William Canfield, Novazyme founder and chief scientific officer.

"The Orphan designation is an important step forward in our path to the human clinic and in providing this treatment to the patients with no current alternative," he said. "We are especially proud to have effectively advanced the development of this technology and to have achieved this level of regulatory recognition so efficiently."

Pompe disease is a fatal neuromuscular disease for which there is currently no approved therapy.

In its application for orphan status, filed in July, Novazyme submitted pre-clinical evidence demonstrating that its highly phosphorylated product for Pompe disease, designated as NZ-1001, provides greatly enhanced enzyme uptake into affected cells, Canfield said. Enhanced enzyme uptake is widely viewed as the key to the treatment of all lysosomal storage diseases, particularly Pompe's.

Orphan drug designation qualifies Novazyme to receive certain benefits such as tax credits and marketing exclusivity from the government.

"With this acknowledgement from the FDA, we are confident that we are well-positioned to move our proprietary technologies rapidly into the human clinic on multiple diseases and to take a leadership position in this therapeutic field," said Anthony McKinney, vice president for drug development.

"If Novazyme's enzyme therapy for Pompe Disease proves effective, this would bring enormous benefits to many Pompe patients worldwide who are in such desperate need for a therapy to relieve their life- threatening disease," said Alfred Slonim, director of the Pompe Disease Center at North Shore University Hospital in Manhasset, N.Y., and an associate professor of pediatrics at New York University.

Novazyme is a pharmaceutical company developing biotherapies for the treatment of lysosomal storage disorders. The biotherapies are based on Novazyme's proprietary technologies for the targeted delivery of the missing enzymes critical for the treatment of these diseases.

The technologies were developed by Canfield in his laboratories at the University of Oklahoma Health Sciences Center. Canfield, an associate professor of medicine at the university, founded Novazyme in 1999. The company's principal investors include Catalyst Health & Technology Partners of Boston; HealthCare Ventures, Princeton, N.J.; the Perseus-Soros Biopharmaceutical Fund, New York; and Neose Technologies.

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