BASEL, Switzerland, Nov. 29, 2018 — Therachon AG (“Therachon”), a clinical-stage biotechnology company focused on treating the root causes of rare conditions, today announced that the European Commission has granted Orphan Drug Designation for apraglutide for the treatment of short bowel syndrome (SBS).
“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, M.D., chief executive officer 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.”
In Europe, 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.
Therachon is a clinical-stage global biotechnology company focused on developing medicines for serious rare conditions with significant unmet medical need. The company is pursuing programs in rare conditions with well characterized biological root causes in both short bowel syndrome (SBS) and achondroplasia. Therachon is committed to making a difference in the lives of patients living with serious rare disorders. For more information, please visit www.therachon.com.
About Short Bowel Syndrome
Short Bowel Syndrome (SBS) results from extensive intestinal resection due to chronic inflammatory bowel disease (IBD), acute events (e.g. mesenteric infarction) 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.
Apraglutide (FE 203799) is a next-generation, synthetic GLP-2 analog that 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.
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