Shire Receives Breakthrough Therapy Designation for Rare Diseases Drugs

US Food and Drug Administration has granted breakthrough therapy designation for two experimental drugs for rare diseases conducted by the biotechnology company Shire.

Shire has produced positive results for the two drugs in its Phase-II trials. One of the drug is budesonide for eosinophilic esophagitis (EoE) and another is maralixibat for progressive familial intrahepatic cholestasis type 2 (PFIC2).

Eosinophilic Esophagitis is a rare immune system disease occurring due to the accumulation of eosinophils, a type of white blood cells, in the lining of the esophagus, resulting in inflammation.

Budesonide has shown positive results against the disease in the phase -II trial. Now, the company is testing the drug in adolescents and adults ages 11-55 years with EoE in the phase-III trial.  


Progressive familial intrahepatic cholestasis type 2 is a group of autosomal-recessive liver disorders developing during infancy. It usually progresses into cirrhosis  The experimental drug maralixibat has reduced serum bile acids, skin itching, leading to normalization of liver in some patients tested in the phase-II trial. 

Source: Medindia

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