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FDA Grants Orphan Drug Status to Coagulin-B for Hemophilia

ALAMEDA, CA -- July 9, 2001 -- Avigen, Inc. announced today that it has received two notices from the U.S. Food & Drug Administration (FDA) that the company's adeno-associated viral (AAV) vectors containing the gene for human coagulation factor IX (Coagulin-B™) qualify for orphan designation. One designation covers the vector for intramuscular delivery and the second covers the vector for liver delivery of its gene therapy for patients with moderate to severe hemophilia B.

The Orphan Drug Act was established by Congress to encourage research and development of new therapies for rare diseases and conditions, i.e. those that affect less than 200,000 patients in the U.S. Under this law, sponsors developing products designated as orphan drugs may receive help through the Orphan Drug Program in facilitating the development of the product and may be entitled to seven years of marketing exclusivity upon final FDA marketing approval.

Currently, Avigen is conducting clinical trials for Coagulin-B at Stanford University Medical Center and The Children's Hospital of Philadelphia, where physicians are testing the safety of both muscle and liver delivery options.

"We are extremely gratified that the FDA has chosen to grant us orphan drug status. Hemophilia B affects approximately 20,000 people worldwide and it's an important first frontier for proving the value of gene therapy. As we endeavor to prove the safety and efficacy of our approach it benefits all members of the gene therapy research community, working toward commercializing a broad range of gene therapy products for the treatment of various diseases," said John Monahan, Avigen's President and Chief Executive Officer.

Physicians and scientists at The Children's Hospital of Philadelphia and Stanford University Medical Center have been conducting a Phase I clinical trial of Avigen's proprietary AAV vector gene therapy for intramuscular delivery of this treatment for hemophilia B. Early results have shown the therapy to be safe and well tolerated in all patients treated with no serious adverse events being noted and indications of low levels of circulating factor IX in the blood of some patients.

In June, Avigen announced the beginning of a second clinical trial for Coagulin-B. This Phase I safety and dose-ranging study will evaluate the administration of Coagulin-B into the patient's hepatic artery and thereby into the liver, where the factor IX protein is normally made. "Our clinical trials are quite straightforward. After treatment with our AAV vectors we simply measure the circulating factor IX in the patient's blood and monitor their on-going need for any supplemental recombinant factor IX injections. There is a good correlation between blood levels of factor IX and clinical outcome," said John Monahan. Avigen hopes to report initial results from this clinical trial, underway at the same medical centers, later this year.

SOURCE: Avigen, Inc.

Related Link: Avigen, Inc.

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