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EU grants Therachon Orphan Drug Designation

05.12.2018 09:30

The European Commission has granted Therachon an Orphan Drug Designation for apraglutide for the treatment of short bowel syndrome (SBS). The clinical stage biotech company is specialised in treating the root causes of rare conditions.

Short Bowel Syndrome (SBS) results from extensive intestinal resection due to chronic inflammatory bowel disease (IBD), acute events or congenital abnormalities. SBS is a severe, chronic condition associated with reduced or complete loss of intestinal function (termed as ‘intestinal failure’). Intestinal failure can be life-threatening and is characterized by malabsorption and malnutrition, infections, blood clots and poor quality of life. Affected individuals are dependent on daily parenteral support, sometimes requiring up to 16 hours of parenteral feeding per day. An estimated 20,000-40,000 patients are thought to suffer from SBS in the US and Europe.

The biotechnology company, Therachon has specialized in developing treatments for rare conditions such as SBS. The company developed Apraglutide (FE 203799), a next-generation, synthetic GLP-2 analog to counter SBS. Apraglutide has undergone extensive preclinical characterization and optimization. It has successfully completed Phase 1 single ascending dose/multiple ascending dose clinical trials in healthy volunteers demonstrating a superior pharmacokinetic profile with a half-life of 30 hours, enabling an easy-to-use, once-weekly dosing regimen. Apraglutide is currently being investigated in two Phase 2 clinical trials in SBS.

Recently, Therachon was granted the EU Orphan Drug Designation for its apraglutide for the treatment of short bowel syndrome (SBS). The orphan drug designation provides regulatory and financial incentives to companies developing products to treat serious rare conditions that affect no more than five in 10,000 persons in the European Union and for which either, no satisfactory treatment exists, or the product candidate would provide a significant benefit for those affected by the condition compared with existing therapies. Incentives include protocol assistance and market exclusivity.

 “We are pleased to receive this designation, which was granted upon reviewing both preclinical study results from animal models and Phase 2 clinical trial data obtained with weekly dosing of apraglutide in patients with SBS,” said Luca Santarelli, CEO of Therachon. “We believe that apraglutide’s orphan drug designation is consistent with its potential to become the best-in-class therapeutic for the treatment of SBS.”

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