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news.Shire has received breakthrough therapy designation from the US Food and Drug Administration (FDA) for two investigational products, SHP621 and SHP625, for rare gastrointestinal (GI) conditions.
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SHP621 (budesonide oral suspension) is in phase 3 development for the treatment of eosinophilic esophagitis (EoE).
EoE is a severe, chronic and rare disease that stems from an elevated number of eosinophils, a type of white blood cell that infiltrate esophagus walls.
It is characterized by an inflammation of the esophagus that could result in difficulty swallowing (dysphagia).
SHP621 met the co-primary endpoints in a phase 2 trial in adolescents and adults (aged 11-40) with EoE.
Compared with placebo, 12 weeks of budesonide oral suspension treatment significantly reduced both dysphagia symptoms and achieved higher proportion of subjects with histologic response.
SHP625 (maralixibat) is currently in phase 2 trials for the treatment of progressive familial intrahepatic cholestasis type 2 (PFIC2), part of a group of autosomal-recessive childhood liver disorders that disrupt bile formation.
Maralixibat inhibits the action of a protein called apical sodium-dependent bile acid transporter.
In a phase 2 single-arm, open-label study, PFIC2 demonstrated decreases from baseline in serum bile acids, marked reductions in pruritus, and normalization of liver parameters in those patients with liver enzymes that were elevated at baseline.
Shire CEO Flemming Ornskov 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."
Image: Shire location in Lexington Massachusetts. Photo: courtesy of John Phelan.