Research & Development

Approach

N-003 is a reprofiling project targeting renal orphan diseases

About drug reprofiling

Over the past few years, well-established marketed drugs have been successfully developed as new treatments for diseases different than those originally intended. This process is called drug repositioning or drug reprofiling. The principal advantage of this process is a significantly shorter and less cost-intensive development for new therapies. As the reprofiled drug is usually on the market for a few years and has undergone regulatory review and approval, it is usually the case that the potential risks of the drug are well known. Reprofiling a drug has a significantly lower rate of failure as significant knowledge about the active substance is available

Orphan Diseases

Traditionally, pharmaceutical companies have focused on developing drugs for large markets. Recently, however, there is an interest in developing drugs for smaller patient populations. In some cases the target patient population is so small, that they are considered Orphan Diseases. These patients usually have few treatment options. In the last decade, considerable interest and success in developing novel treatments for these areas of unmet medical need has been achieved.