Hurdles in drug discovery for Gaucher’s disease

Abstract

Metabolic disorders are increasing day by day in current world population. The percentage of the metabolic disorder in US population is less than 20,000 so it has been considered as an orphan disease. For development of drug to an orphan disease is a challenging task for the pharmaceutical industries because of various factors. Among all the metabolic disorder Gaucher’s disease is the one rarely seen condition in the people so has been considered as an orphan disease. Medication for the orphan disease is called as orphan drug gauchers disease can be seen in children and adults but, as per the researches the impact of disease is more in children than that of the adult. Another problem with gauchers disease is misdiagnosis of the condition. Main problem in the drug discovery for orphan drug in the EU and FDA is mainly because of lack of harmonization in the orphan drug regulations between FDA and EMA. Another major huddle for the discovery of the medication for the orphan disease like gaucher’s disease is lack of synchronization and appropriate guidance from the regulatory industry and this will be burden for the sponsor’s to manufacture a medication for orphan disease like Gaucher’s disease.