BASEL, Switzerland, Jan. 16, 2019 — Therachon AG (“Therachon”), a clinical-stage biotechnology company focused on the discovery, development and commercialization of innovative treatments for serious rare conditions, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for apraglutide for the treatment of short bowel syndrome (SBS).
“We are pleased to receive this designation in the U.S. and look forward to advancing this potentially best-in-class treatment option for people in the U.S. living with short bowel syndrome,” said Luca Santarelli, M.D., chief executive officer of Therachon. “We see this as another important milestone in our quest to improve upon the current standard-of-care for patients with SBS.”
In the U.S., Orphan Drug Designation provides orphan status to investigational therapies aimed to treat rare diseases and disorders affecting fewer than 200,000 people, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. Investigational therapeutics with orphan status qualify for a number of incentives aimed to advance clinical development.
Apraglutide has also received Orphan Drug Designation from the European Commission.
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, including both short bowel syndrome 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, known as ‘intestinal failure’. Intestinal failure caused by SBS can be life-threatening and is characterized by malabsorption and malnutrition. Affected individuals are dependent on daily parenteral support, typically requiring between 10 – 15 hours of parenteral feeding per day. Parenteral support is associated with infections, blood clots and poor quality of life. An estimated 20,000-40,000 patients are thought to suffer from SBS in the U.S. 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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