Deputy Director of the Office of Drug Evaluation III of the FDA's Center for Drug Evaluation and Research, Amy G. Egan, M.D., M.P.H., announced the approval of a treatment drug called Cerdelga for adult patients with Type 1 Gaucher disease.
A patient with Gaucher disease have cells that are unable to produce enough enzyme called glucocerebrosidase, which aids in breaking down a fatty substance called glucocerebroside. If the body is low in glucocerebrosidase, the fatty substance builds up and enlarges the cells. This enlargement is called Gaucher disease.
Gaucher disease is reportedly a rare disease; it is estimated to affect about 1 in 50,000 to 1 in 100,000 people worldwide. It frequently occurs to those of Eastern and Central European Jewish decent, named Ashkenazi, approximately affecting 1 in 400 to 1 in 600 people, reported Phizer.
Gaucher is a disease that has no cure. However, long term treatment is now readily available to help manage the disease.
Cerdelga is an oral treatment drug, produced by the first producers for Gaucher's treatment 20 years ago. It is reportedly the best alternative treatment of the intravenous drug Cerezyme, which averagely priced at 300,000 USD for the annual treatment.
FDA, committed to protect the public's health by assuring the safety and effectiveness of drugs manufactured by companies, aims to supply the public with FDA stamped drugs to help treat rare diseases like Gaucher disease.
