부산대학교 도서관

Enzyme replacement therapy (ERT) is the f rst-line treatment for Gaucher disease (GD). The ERT Cerezyme (imiglucerase) was approved by the US Food and Drug Administration (FDA) in 1994; however, its patents have expired allowing the development of non-originator biological drugs. The Group of Medical Specialists on Gaucher disease in Mexico claims there is a 'regulatory gap' in the def nition of biosimilar orphan drugs which should be resolved before these drugs are approved and marketed. In Mexico, Asbroder (imiglucerase; Abcertin in other countries) has been approved for the treatment of GD, but it does not meet the exact def nition of a biosimilar described by the World Health Organization (WHO) and other international guidelines. However, it was recognized as an orphan drug for GD and approved by the medicines regulatory authority in Mexico with the same international non- proprietary name (imiglucerase), in order to increase national access to treatment. This regulatory practice opens up a debate surrounding the relationship between international guidelines, clinical medicine, scientif c evidence, bioethical considerations, public health institutions, and local laws. The aim of this paper is to establish whether there are biosimilars ofimiglucerase as a therapeutic option. Moreover, it describes the regulatory setting of non-originator biological drugs in orphan diseases based on the approval of the non-originator imiglucerase in Mexico. Keywords: Biosimilar, Gaucher disease, imiglucerase, non-originator imiglucerase, orphan drugs
Introduction Orphan drugs are biological or chemical drugs used for the prevention, diagnosis or treatment of rare diseases affecting no more than five in every 10,000 inhabitants [1-3]. Biological drugs [...]