Biotechnology company Shire has announced that the Scottish Medicines Consortium (SMC) has accepted its Revestive® (teduglutide) 5mg and 1.25mg for restricted use in the treatment of children aged one year and above with short bowel syndrome (SBS).
This disabling condition is caused by an inability to absorb sufficient nutrients and fluids to survive and grow. Patients should be stable following a period of intestinal adaptation after surgery.1
Shire said the decision means children and young people up to the age of 17 years in Scotland will be the first in the UK to benefit from a treatment that has the potential to reduce their reliance on parenteral support (PS) – parenteral nutrition (liquid food) and/or intravenous fluids, which contains all their nutritional and fluid needs and is given directly into the blood.
Teduglutide – a glucagon-like peptide-2 (GLP-2) analogue – is the first and only licensed pharmacological therapy that addresses short bowel syndrome with intestinal failure (SBS-IF), by enhancing gut adaptation through improving absorption of nutrients and fluids. This allows patients to reduce, and in some cases, even wean off PS.2
An open label clinical study demonstrated that after 12 weeks of teduglutide treatment (0.05mg/kg/day), 20% (3/15) of children became independent of PS, and the remainder saw a trend in reductions in PS volume. In the standard of care (SOC) arm (n=5), none of the children became independent of PS, and there was no change in PS volume.
Because of the small pool of eligible patients, this study analysis was only descriptive and its limitations included being short-term, with an open label design, and a small sample size.
Commenting on the SMC decision, Damien Bailly, UKIE cluster head and general manager UK, said: “At Shire, we are focused on bringing true innovation to areas where we can have a significant impact on patients with high unmet need.
“We welcome the SMC decision and its rapid implementation so children can benefit as soon as possible, andwe remain committed to working with the SMC to secure access to this therapy for adults. We would like to thank the patient groups, carers and clinicians who have all supported our SMC submission.”
The National Institute for Health and Care Excellence (NICE) is appraising teduglutide for the treatment of patients aged one year and above with SBS in England, and is expected to issue final guidance later in 2018.
- Revestive® EMA Summary of Product Characteristics, Shire Pharmaceuticals Ireland Limited. Available at: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/002345/WC500132926.pdf
- NICE, 2017. Appraisal consultation document. Teduglutide for treating short bowel syndrome.