Orphan drugs and pharmacogenomics

PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Orphan drug designation and pharmacogenomics: options and opportunities” (BioDrugs. 2006;20(2):71-79). Authors are Maher PD and Haffner M from the Office of Orphan Products Development, Food and Drug Administration, Maryland, USA. The rapid increase in characterization and understanding of the human genome has had a major impact on the development of therapies for rare diseases. The “inborn errors of metabolism”, which are generally rare diseases, are beginning to realize new therapies based on an understanding of disease processes at the genetic level. Likewise, an understanding of acquired genetic errors, as seen in cancer, is allowing for targeted approaches to therapy that are revolutionizing, in many cases, both standards of care and prognosis. With the recent completion of sequencing of the human genome, as well as the increasing ease of DNA sequencing, the promise and challenge of the pharmacogenetic approach to treatment will be expected to play an increasingly important role in development of new therapies for both rare and common diseases. To access the full abstract of the article, click here.