Our Story

The story of Rare Disease Therapeutics, Inc., is the story of hope vs. reality. We specialize in filling very specific and special medical needs of a small population of adults and children seeking life-preserving therapies for rare diseases. Many of these diseases are not immediately recognizable to even the most astute physicians; therefore, the role of Rare Disease Therapeutics, Inc., must include creating awareness of such diseases and their diagnosis. Unfortunately, the reality is that all too few companies are willing to invest in low volume, low return research programs. Thus the needs of the few are lost to corporate profit/loss statements.

Rare Disease Therapeutics, Inc., on the other hand, is a company built on the hope and premise of providing options where there are no options, and providing therapies where there are no therapies, through creative strategic alliances, as well as unique relationships with the government, research institutions, physicians, scientist, and manufacturers.

Rare Disease Therapeutics, Inc., is a well established company founded in 1991 as Orphan Pharmaceuticals, U.S.A., Inc., strategically located in Nashville, Tennessee. The company changed its name to Rare Disease Therapeutics, Inc., in March 2001 to better describe its mission. Rare Disease Therapeutics, Inc., has a solid track record with multiple licensing agreements, a comprehensive global patient advocacy network, and significant success in drug development and approval.

The company’s major clinical expertise and licensing focus have been in rare diseases in the following therapeutic areas:

Genetic disorders
Metabolic diseases
Hepatic diseases

In addition to the above cited therapeutic areas, Rare Disease Therapeutics, Inc., is also seeking current orphan products or low volume mature products that would be considered as orphans if these products were launched today.

What are “Orphan Drugs”?

Orphan products are drugs, biologics, or other therapeutics that treat diseases that affect fewer than 200,000 people in the United States.

What is the Orphan Drug Act of 1983?

The U.S. Orphan Drug Act (1983) gives incentives to pharmaceutical companies to develop drugs, biologics, or other therapeutics that will treat diseases that affect fewer than 200,000 people in the United States. The orphan drug law offers tax breaks and a seven-year exclusivity on product sales to induce companies to undertake the development and manufacturing of such product, which otherwise might not be profitable because of the small potential market. (Of the 5,000 diseases covered under the act, 47% affect fewer than 25,000 people.). The law has led to the introduction of over 200 valuable new products for the treatment of rare diseases.

America's First
Exclusively Orphan
Drug Company