Orphan drug status for apraglutideMarch 7, 2019
T herachon AG said the US FDA granted Orphan Drug Designation for apraglutide for the treatment of short bowel syndrome (SBS).
Apraglutide is a glucagon-like peptide-2 receptor agonist.
SBS results from extensive intestinal resection due to chronic inflammatory bowel disease (IBD), acute events such as 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.
In the US, Orphan Drug Designation provides orphan status to investigational therapies aimed to treat rare diseases and disorders affecting fewer than 200,000 people.
Therachon is a clinical-stage global biotechnology company pursuing programmes in rare conditions with well-characterized biological root causes, including both short bowel syndrome and achondroplasia.