Does Teduglutide work?
Yes, in a controlled trial plus a two-year follow-up of 88 patients.
Approval rests on the randomized placebo-controlled STEPS trial in adults with short bowel syndrome, followed by a two-year open-label extension (STEPS-2) in 88 patients that documented sustained reductions in parenteral support. The approved use matches the studied use, so it grades A. The outcome measured is volume of intravenous nutrition, not survival. 12
The STEPS trial lasted 24 weeks and included 86 adults. A 20 percent or greater reduction in weekly parenteral support volume was the primary measure and the basis for approval. 63 percent on teduglutide reached it, versus 30 percent on placebo. 2
STEPS-2 was the two-year open-label extension, published in Clinical and Translational Gastroenterology. It followed 88 adults from 25 centers in Europe and North America. 30 patients were treated for the full 30 months. Of these, 28 (93 percent) kept at least a 20 percent reduction in parenteral support. Weekly infusion volume fell by more than half on average. A minority of patients came off parenteral support entirely. Patients who entered the extension from the placebo arm also responded once started on the drug. 1
Evidence checklist
- Randomised controlled trial STEPS placebo-controlled trial
- Human study over 100 people STEPS-2 extension had 88 patients
- More than one research group Trials run by the developer
- Clinical outcomes, not surrogate markers Intravenous volume, a proxy outcome
- Human safety data beyond case reports Trial and label safety data
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Citations
- Schwartz 2016, Clin Transl Gastroenterol: Long-term teduglutide for intestinal failure with short bowel syndrome (STEPS-2) doi.org
- FDA 2021: Gattex (teduglutide) prescribing information accessdata.fda.gov
Reference information only, not medical advice. Drawn from the Teduglutide article, fact-checked against the cited sources. No dosing anywhere on this site.