The Food and Drug Administration (FDA) has granted Orphan Drug designation to apraglutide (Therachon AG), an investigational treatment for short bowel syndrome (SBS).
The Company has already completed a Phase 1 trial of this synthetic glucagon-like peptide-2 (GLP-2) analog in healthy volunteers. Two Phase 2 trials in SBS patients with intestinal failure and insufficiency are currently underway in which a once-weekly dosing regimen will be evaluated
“We see this [FDA Orphan Drug designation] as another important milestone in our quest to improve upon the current standard-of-care for patients with SBS,” said Luca Santarelli, MD, CEO of Therachon.
Short bowel syndrome is a severe and chronic condition associated with reduced or complete loss of intestinal function, often resulting from extensive intestinal resection associated with inflammatory bowl disease, vascular events (embolism/thrombosis), congenital abnormalities, malignancy, and other causes (i.e., complications of bariatric surgery, strangulated hernia). In 2012, the FDA approved Gattex (teduglutide; NPS), a GLP-2 analog, for the treatment of adult patients with SBS who are dependent on parenteral support.