Shire gets FDA breakthrough therapy designation for two rare disease drugs
pharmafile | June 13, 2016 | News story | Manufacturing and Production, Research and Development, Sales and Marketing | Breakthrough Therapy Designation, Shire, US FDA, rare disease, rare disease drugs
Ireland headquartered Shire (LSE: SHP) on Monday said the US Food and Drug Administration (FDA) has granted breakthrough therapy designation for two of its rare disease trial drugs.
The two compounds include SHP621 (budesonide oral suspension, or BOS) for eosinophilic esophagitis (EoE), and SHP625 (maralixibat) for progressive familial intrahepatic cholestasis type 2 (PFIC2).
Flemming Ornskov, chief executive of Shire, said: “Receiving Breakthrough Therapy Designation on two pipeline products this past week reflects the potential of our strong and innovative pipeline of more than 60 programs.”
The EoE 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 about 15-55 cases per 100,000 persons, with high-end estimates reported by studies in Western regions.
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.
FDA’s breakthrough therapy designation is granted to a therapy that is intended to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over current standard of care.
Anjali Shukla
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