Pompe Disease Therapy Receives Orphan Designation

, Inc. has received formal notification from the Office of Orphan Products Development at the FDA that its proprietary, highly phosphorylated enzyme replacement therapy for the treatment of Pompe Disease has been granted orphan drug status.

This notification from the FDA represents the first official validation of Novazyme‘s core phosphorylation technology platform with which the Company intends to address the unmet needs of lysosomal storage disease (LSD) patients.

In its application for orphan status, filed on July 14, 2000, 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.

Enhanced enzyme uptake is widely viewed as the key to the treatment of all lysosomal storage diseases, particularly Pompe’s. With more efficient uptake of replacement enzymes, patients may benefit by greater response to these therapies and by reduced side effects, such as antibody responses.

The company’s filing also highlighted the fundamentally different molecular structure of a lysosomal enzyme replacement therapy that is highly phosphorylated and that lacks complex carbohydrate structures.

The FDA notification specifically stated that: “We have determined that [Novazyme’s] recombinant human highly phosphorylated acid alpha- glucosidase (rhHPGAA) qualifies for orphan designation for enzyme replacement therapy in patients with all subtypes of glycogen storage disease type II.”

“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.

We are especially proud to have effectively advanced the development of this technology and to have achieved this level of regulatory recognition so efficiently,” commented Dr. William Canfield, Novazyme founder and Chief Scientific Officer.

Novazyme is a pharmaceutical company developing biotherapies for the treatment of lysosomal storage disorders.

These biotherapies are based on Novazyme’s proprietary technologies for the targeted delivery of the missing enzymes critical for the treatment of these diseases.

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