13 June 2016 - Shire today announced that the U.S. FDA has granted breakthrough therapy designation for two investigational products for rare diseases: SHP621 (budesonide oral suspension) for eosinophilic oesophagitis and SHP625 (maralixibat) for progressive familial intrahepatic cholestasis type 2.

Eosinophilis oesphagitis is a serious, chronic and rare disease that stems from an elevated number of eosinophils, a type of white blood cell, that infiltrate the walls of the esophagus. EoE is characterized by an inflammation of the esophagus that may lead to difficulty swallowing (dysphagia). The diagnosed prevalence of EoE ranges from approximately 15-55 cases per 100,000 persons, with high-end estimates reported by studies in Western regions.

Pprogressive familial intrahepatic cholestasis (PFIC) refers to a group of autosomal-recessive liver disorders of childhood that disrupt bile formation and present with cholestasis. The symptoms of PFIC include severe itching of the skin (pruritus), and jaundice. PFIC is estimated to affect 1 in 50,000 to 1 in 100,000 births. PFIC2 is the most common type of PFIC, accounting for around half of cases.

For more details, go to: https://www.shire.com/newsroom/2016/june/13-06-2016-shire-receives-fda-breakthrough-therapy-designation