The Official Website of the Acid Maltase Deficiency Association

It is difficult to say what is impossible for the dreams of yesterday are the hopes of today and the reality of tomorrow. - Robert H. Goddard

Duke University Starts Clinical Trials for Pompe’s Disease

Posted on: June 30, 1999

The following status statement was released by Synpac (North Carolina), Inc., on June 30, 1999

The following status statement was released by Synpac (North Carolina), Inc., on June 30, 1999

Synpac Pharmaceuticals Limited
Cambois, Bedlington, Northumberland NE22 7DB
Tel: (01670) 565656 Fax: (01670) 850571

I.S. Hodgson-Direct Line
Tel: 01670 565539
Fax: 01670 522459

On May 24, 1999, (see press release above article), Duke University announced the start of clinical trials to test the safety and efficacy of recombinant human acid alpha-glucosidase (rhGAA) for the treatment of glycogen storage disease type II (Pompe Disease). The initial study will include up to three infantile patients who meet the inclusion criteria defined in the study protocol. Dr. Y.T. Chen, Chief of Medical Genetics at Duke, is the sponsor of the study. Synpac (N.C.), Inc., is funding the study and has provided the rhGAA.

Because it is known that individuals with Pompe disease lack the acid alpha-glucosidase enzyme, it has been proposed that enzyme replacement therapy with rhGAA would treat the symptoms related to Pompe disease, much like synthetic insulin can treat the symptoms of individuals with insulin dependent diabetes. When rhGAA was administered to an animal model of Pompe disease (Japanese quail that lack the same enzyme and have similar clinical features of Pompe disease), significant improvement in muscle strength was observed after three weeks. It is anticipated that if Pompe patients are treated with a manufactured version of acid alpha- glucosidase (rhGAA), the symptoms of Pompe disease may be alleviated. The current clinical trial is designed to test the safety and effectiveness of this enzyme in humans with Pompe disease.

It is important to note that rhGAA is not designed to be a cure for Pompe disease. Rather it is intended as a therapeutic agent to treat Pompe disease. The rhGAA will be administered intravenously to supplement the body’s insufficient supply of enzyme. It is anticipated that patients will require life long therapy with rhGAA.

Synpac plans to expand the trial to include juveniles and adults and to expand the trials to Europe; however, the timing and design of all phases of the clinical trials depend upon a number of factors including:

Analysis of results from the initial trial.
Questions that this first study is designed to answer include: Is the drug well tolerated? Are there side effects? Is there evidence of clinical benefit? In future studies, data will be generated to determine what the effective dose will be and how frequently it will need to be administered.

Review and permission of regulatory agencies.
The Food and Drug Administration (FDA) must review the drug safety and tolerance data generated during this first phase and grant permission to expand the number patients. All efforts are directed at moving this product through the regulatory requirements as quickly as possible, while assuring the safety and clinical benefit of the product. Advantage will be taken of the provisions for rapid approval through the FDA.

Continued manufacturing success
Manufacturing Plans: Manufacturing efforts must continue to be successful as we expand production to meet anticipated demands. Although the trials have just begun, Synpac has already started developing a full scale manufacturing program to generate a drug supply sufficient for the anticipated market of infants, juveniles, and adults. We anticipate the clinical trials will progress in parallel with the manufacturing efforts to increase supply.

Manufacturing Methods: To manufacture the rhGAA, Synpac is using a Chinese hamster ovary (CHO) cell line, which Dr. Chen’s laboratory at Duke genetically engineered to contain the human GAA gene. As the cells grow in a nutrient rich broth, they secrete the rhGAA enzyme into the broth. The enzyme is then purified from the broth through a series of filtering and sieving devices. CHO cell technology is not new and has been used for many years to manufacture a number of medical products on the market.

Manufacturing Capabilities: Synpac initially produced a preliminary drug supply in a small pilot manufacturing facility. However, Synpac has now moved its manufacturing efforts to an established contractor with large scale manufacturing capabilities. By using a contract manufacturer, Synpac avoids the major investments of both time and costs required to build a manufacturing facility and gains the expertise of a team of scientists who have prior experience manufacturing similar proteins.

“It is our goal to insure a continuous drug supply to all patients enrolled in clinical trials.”

Disclaimer: The AMDA does not endorse any of the products, medications, treatments or information reported herein. The website and its contents is intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.

Address: AMDA PO Box 700248
San Antonio, Texas 78270 USA