Title: FDA Grants Orphan Drug Status to Oral Human Gammaglobulin for Juvenile Rheumatoid Arthritis

URL: http://www.pslgroup.com/dg/1FD936.htm

Doctor's Guide
June 8, 2001
TUCSON, AZ -- June 8, 2001 -- Protein

Therapeutics Inc. announced yesterday that it has received orphan drug designation from the United States Food and Drug Administration (FDA) for the use of oral human gammaglobulin for the treatment of juvenile rheumatoid arthritis (JRA). This is the first indication for the use of oral human immunoglobulin. Orphan drug designation entitles Protein Therapeutics to exclusive marketing rights in the United States for seven years following market approval and enables the company to apply for research funding, tax credits for certain research expenses, and a waiver from the FDA's application user fee.

In the past, orphan drug designation has been associated with other biopharmaceuticals such as Avonex(R), Epogen(R) and Neupogen(R). Although JRA is the first indication for which Protein Therapeutics has sought orphan drug status, it is by no means the only disease for which oral human gammaglobulin may be appropriate.

Supplementing the intestinal tract with pooled immunoglobulin has a dramatic impact on the course of JRA, and this discovery may open the door to studies of its use in a variety of related diseases with autoimmune characteristics.

"The FDA's Orphan Drug Program provides a number of benefits to Protein Therapeutics," said Protein Therapeutics president Dr. Leon Barstow. "Oral gammaglobulin for the treatment of JRA is a good example of why the orphan drug program was created. We are very pleased to be able to take advantage of this innovative FDA program."

Juvenile rheumatoid arthritis is a systemic inflammatory disease accompanied by symmetrical polyarthritis commonly affecting the small joints of the hands and feet. Both American and European criteria for classifying the disease include children from birth to 16 years of age and three major onset types: systemic, pauciarticular, and polyarticular.

JRA is currently treated using a combination of potent disease-modifying anti-rheumatic drugs (DMARDs) such as methotrexate, corticosteroids and cyclosporine.

Recently, new therapies aimed at the specific reduction of inflammatory cytokines such as tumor necrosis factor alpha (TNF-a) have been introduced, however, combinations of these drugs are usually required for adequate control of disease activity.

The use of corticosteroids and immunosuppressants is associated with significant risk and side effects, both in terms of toxicity and vulnerability to serious infections.

Currently there exists an urgent need for new, safe methods for treating JRA that do not entail the side effects associated with conventional therapy.

Oral gammaglobulin is a sterilized immune globulin product prepared from pooled normal human donor plasma that consists primarily of IgG. If proven effective in clinical trials currently underway it will be a safe, nontoxic treatment for JRA.