Orphan

In this episode Lucy Chard is joined by Rajiv Khatau to discuss the importance of looking into new therapeutic areas and some of the more niche areas of pharmaceuticals, and investing in the future of the industry. 

Enzyme replacement therapeutic Xenpozyme has received US FDA approval with an orphan drug designation to treat symptoms of Acid Sphingomyelinase Deficiency, a rare genetic disease.

In France, rare diseases are numerous: between 7,000 and 8,000 diseases are currently accounted for, and concern more than 3 million French people, accounting for almost 4,5% of the total population. The organization of screening and care for these ill...