Shire announced that the Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for two investigational drugs for rare diseases: SHP621 (budesonide oral suspension, or BOS) for eosinophilic esophagitis (EoE), and SHP625 (maralixibat) for progressive familial intrahepatic cholestasis type 2 (PFIC2).

EoE is a serious, chronic and rare disease characterized by an inflammation of the esophagus due to elevated eosinophils, which may lead to dysphagia. PFIC2 is the most common type of PFIC, a group of childhood autosomal-recessive liver disorders characterized by cholestasis due to the disruption of bile formation. There are currently no pharmacological treatment options indicated for either EoE or PFIC. 

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The Breakthrough Therapy designation for BOS was supported by data from a Phase 2 trial in patients with EoE. The study met both co-primary endpoints, demonstrating significant reduction of dysphagia symptoms and higher proportion of patients with histologic response for patients treated with budesonide oral suspension vs. placebo. SHP621 BOS is currently in a Phase 3 clinical trial.

Maralixibat was granted Breakthrough Therapy designation based on results from the Phase 2 trial, INDIGO, in pediatric patients with PFIC2. Although the interim results demonstrated no statistical significance in the reduction of mean serum bile levels from baseline across the study population as a whole, a subpopulation of patients showed decreases in serum bile acids from baseline, marked reductions in pruritus, and normalization of liver parameters in patients with elevated baseline liver enzymes. Maralixibat is currently in Phase 2 trials for PFIC.

BOS is a novel, topically active, oral viscous formulation of budesonide specifically formulated for EoE. Maralixibat is a potent, oral, selective inhibitor of the apical sodium-dependent bile acid transporter (ASBT) designed to block bile acid reabsorption in the ileum and increases fecal bile acid excretion.

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