ZUG, Switzerland I May 27, 2016 I Shire plc (LSE: SHP, NASDAQ: SHPG) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the extension of the approval of Revestive®* (teduglutide) 5 mg powder and solvent for solution for injection in paediatric patients (aged one to 17 years) with short bowel syndrome (SBS). Teduglutide is an analogue of human glucagon like peptide 2 (GLP-2) that enhances key structural and functional adaptations in the intestinal mucosa.i
The European Commission (EC) will now consider the CHMP positive opinion in its final decision of whether to extend marketing authorisation for teduglutide in paediatric patients with SBS in the European Union; a final decision from the EC is expected in August 2016.
“We are delighted that the CHMP has rendered a favorable recommendation for teduglutide in paediatric patients based on their evaluation of the data and benefit-to-risk balance,” said Philip J. Vickers, Ph.D., Head of Research and Development, Shire. “We await the final decision of the EMA and the potential to bring a new treatment option for children and adolescents suffering from SBS in Europe.”
SBS is a rare gastrointestinal condition characterised by a clinically significant reduction in intestinal absorptive capacity as a consequence of surgical resection of large portions of the intestine, commonly due to congenital abnormalities, disease or trauma.vi, vii, viii
Revestive is currently indicated in Europe for the treatment of adult patients with SBS, who should be stable following a period of intestinal adaptation after surgery, i in Canada for the treatment of adult patients with SBS who are dependent on parenteral support (PS),ii and in the United States under the name GATTEX® (teduglutide [rDNA origin]) for injection for the treatment of adult patients with SBS who are dependent on PS.iii
Supportive Paediatric Data
A 12-week, open-label, multicentre, safety, pharmacokinetic and pharmacodynamic study was conducted in 42 children aged 1-17 years who had SBS with Intestinal Failure (SBS-IF) for at least one year and had plateaued in PS reduction with minimal or no advance in enteral nutrition for at least three months. Of the participants, 37 received teduglutide 0.05 mg/kg/day (n=15); 0.025 mg/kg/day (n=14); 0.0125 mg/kg/day (n=8); and five received standard of care. iv, v
Of the 42 patients, 40 (95%) completed the study. Most adverse events were related to gastrointestinal complaints and/or central line-related issues. No deaths were reported, no serious drug-related adverse events were observed, and no patient discontinued the study due to adverse events. No safety signals related to fluid overload, obstruction, hepatobiliary system, or colonic polyps were seen in the study.iv, v
*Revestive and GATTEX are registered trademarks of NPS Pharmaceuticals Inc., part of the Shire Group of Companies
Although the study was not powered for efficacy, the data showed that children treated with teduglutide 0.05 mg/kg/day and 0.025 mg/kg/day had reductions from baseline to week 12 in PS volume requirements, and increases from baseline in enteral nutrition volume. Four patients achieved independence from PS (three in the teduglutide 0.05 mg/kg/day group, one in the 0.025 mg/kg/day group). Despite PS reductions, clinical and nutritional status remained stable across the teduglutide treatment groups. iv, v
About Short Bowel Syndrome
SBS is a rare and potentially life-threatening gastrointestinal condition. It is characterized by a clinically significant reduction in intestinal absorptive capacity as a consequence of surgical resection of large portions of the intestine commonly due to congenital abnormalities, disease or trauma. If intestinal adaptation is inadequate, the absorptive capacity of the residual intestine becomes insufficient to meet the nutritional, fluid and electrolyte needs to sustain the life and growth requirements of a child; this leads to Intestinal Failure (IF), which requires chronic dependence on PS to maintain adequate growth, hydration, protein, electrolyte, and micronutrient balances. vi, vii, viii SBS is the most common cause of IF in the paediatric population.vi, viii
Estimates of the prevalence of SBS-IF in children vary markedly, largely due to the lack of standardised reporting and rarity of the disease.vi, viii In a recent cross-sectional study in the Netherlands (the nationwide DRIFT registry study), the prevalence of chronic IF requiring home PS was 9.6 per million in children.ix In the DRIFT registry population, 43.2% of children with chronic IF had SBS, which translates into a Dutch national prevalence of paediatric SBS-IF requiring home parenteral nutrition of 4.1 per million.ix
Safety Information
This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.
Adverse reactions
Very common adverse reactions (frequency ≥1/10) with Revestive treatment are abdominal pain and distension, respiratory tract infections, nausea, injection site reactions, headache, vomiting, oedema peripheral and gastrointestinal stoma complications.
Common adverse reactions (≥1/100 to <1/10) are influenza, decreased appetite, anxiety, sleep disorder, paraesthesia, cardiac failure congestive, flushing, dyspnoea, cough, pancreatitis, intestinal obstruction, cholestasis and cholecystitis, dermatitis allergic, arthralgia, renal colic, costovertebral angle tenderness, chest pain, night sweats, C-reactive protein increased.
Uncommon adverse reactions (≥1/100 to <1/10) are syncope.
Contraindications
Hypersensitivity to the active substance or to any of the excipients, or trace residues of tetracycline; active or suspected malignancy; history of malignancies in the gastrointestinal tract including the hepatobiliary system within the last five years.
Special warnings and precautions for use
Colo-rectal polyps; gastrointestinal neoplasia including hepatobiliary tract; gallbladder and bile ducts; pancreatic diseases; monitoring of small bowel, gallbladder and bile ducts, and pancreas; intestinal obstruction; cardiovascular; management of fluids during treatment with Revestive; concomitant medication; special clinical conditions; hepatic impairment; discontinuation of treatment; excipients.
For the current Revestive EU Summary of Product Characteristics, please click here. For the Revestive Product Monograph (Canada), please click here. For the GATTEX U.S. Prescribing Information, please click here.
Shire enables people with life-altering conditions to lead better lives.
Our strategy is to focus on developing and marketing innovative specialty medicines to meet significant unmet patient needs.
We focus on providing treatments in Rare Diseases, Neuroscience, Gastrointestinal and Internal Medicine and we are developing treatments for symptomatic conditions treated by specialist physicians in other targeted therapeutic areas, such as Ophthalmics.
www.shire.com
Date of preparation: May 2016
INTSP/C-ANPROM/TED/16/0036
S12179
iv Carter BA, et al. Clin Nutr 2015;34(Suppl 1):S239.
v Carter BA, et al. J Pediatr Gastroenterol Nutr 2015;61(Suppl 2):S46-47.
vi Wales PW, Christison-Lagay ER. Semin Pediatr Surg 2010; 19(1): 3-9.
vii Sulkowski JP, Minneci PC. Pathophysiology 2014;21(1):111-118.
viii Kaufman SS, Matsumoto CS. Minerva Pediatr 2015;67(4):321-340.
ix Neelis EG, Roskott AM, Dijkstra G, et al. Clin Nutr 2016;35(1):225-229.
SOURCE: Shire
Post Views: 19
ZUG, Switzerland I May 27, 2016 I Shire plc (LSE: SHP, NASDAQ: SHPG) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the extension of the approval of Revestive®* (teduglutide) 5 mg powder and solvent for solution for injection in paediatric patients (aged one to 17 years) with short bowel syndrome (SBS). Teduglutide is an analogue of human glucagon like peptide 2 (GLP-2) that enhances key structural and functional adaptations in the intestinal mucosa.i
The European Commission (EC) will now consider the CHMP positive opinion in its final decision of whether to extend marketing authorisation for teduglutide in paediatric patients with SBS in the European Union; a final decision from the EC is expected in August 2016.
“We are delighted that the CHMP has rendered a favorable recommendation for teduglutide in paediatric patients based on their evaluation of the data and benefit-to-risk balance,” said Philip J. Vickers, Ph.D., Head of Research and Development, Shire. “We await the final decision of the EMA and the potential to bring a new treatment option for children and adolescents suffering from SBS in Europe.”
SBS is a rare gastrointestinal condition characterised by a clinically significant reduction in intestinal absorptive capacity as a consequence of surgical resection of large portions of the intestine, commonly due to congenital abnormalities, disease or trauma.vi, vii, viii
Revestive is currently indicated in Europe for the treatment of adult patients with SBS, who should be stable following a period of intestinal adaptation after surgery, i in Canada for the treatment of adult patients with SBS who are dependent on parenteral support (PS),ii and in the United States under the name GATTEX® (teduglutide [rDNA origin]) for injection for the treatment of adult patients with SBS who are dependent on PS.iii
Supportive Paediatric Data
A 12-week, open-label, multicentre, safety, pharmacokinetic and pharmacodynamic study was conducted in 42 children aged 1-17 years who had SBS with Intestinal Failure (SBS-IF) for at least one year and had plateaued in PS reduction with minimal or no advance in enteral nutrition for at least three months. Of the participants, 37 received teduglutide 0.05 mg/kg/day (n=15); 0.025 mg/kg/day (n=14); 0.0125 mg/kg/day (n=8); and five received standard of care. iv, v
Of the 42 patients, 40 (95%) completed the study. Most adverse events were related to gastrointestinal complaints and/or central line-related issues. No deaths were reported, no serious drug-related adverse events were observed, and no patient discontinued the study due to adverse events. No safety signals related to fluid overload, obstruction, hepatobiliary system, or colonic polyps were seen in the study.iv, v
*Revestive and GATTEX are registered trademarks of NPS Pharmaceuticals Inc., part of the Shire Group of Companies
Although the study was not powered for efficacy, the data showed that children treated with teduglutide 0.05 mg/kg/day and 0.025 mg/kg/day had reductions from baseline to week 12 in PS volume requirements, and increases from baseline in enteral nutrition volume. Four patients achieved independence from PS (three in the teduglutide 0.05 mg/kg/day group, one in the 0.025 mg/kg/day group). Despite PS reductions, clinical and nutritional status remained stable across the teduglutide treatment groups. iv, v
About Short Bowel Syndrome
SBS is a rare and potentially life-threatening gastrointestinal condition. It is characterized by a clinically significant reduction in intestinal absorptive capacity as a consequence of surgical resection of large portions of the intestine commonly due to congenital abnormalities, disease or trauma. If intestinal adaptation is inadequate, the absorptive capacity of the residual intestine becomes insufficient to meet the nutritional, fluid and electrolyte needs to sustain the life and growth requirements of a child; this leads to Intestinal Failure (IF), which requires chronic dependence on PS to maintain adequate growth, hydration, protein, electrolyte, and micronutrient balances. vi, vii, viii SBS is the most common cause of IF in the paediatric population.vi, viii
Estimates of the prevalence of SBS-IF in children vary markedly, largely due to the lack of standardised reporting and rarity of the disease.vi, viii In a recent cross-sectional study in the Netherlands (the nationwide DRIFT registry study), the prevalence of chronic IF requiring home PS was 9.6 per million in children.ix In the DRIFT registry population, 43.2% of children with chronic IF had SBS, which translates into a Dutch national prevalence of paediatric SBS-IF requiring home parenteral nutrition of 4.1 per million.ix
Safety Information
This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.
Adverse reactions
Very common adverse reactions (frequency ≥1/10) with Revestive treatment are abdominal pain and distension, respiratory tract infections, nausea, injection site reactions, headache, vomiting, oedema peripheral and gastrointestinal stoma complications.
Common adverse reactions (≥1/100 to <1/10) are influenza, decreased appetite, anxiety, sleep disorder, paraesthesia, cardiac failure congestive, flushing, dyspnoea, cough, pancreatitis, intestinal obstruction, cholestasis and cholecystitis, dermatitis allergic, arthralgia, renal colic, costovertebral angle tenderness, chest pain, night sweats, C-reactive protein increased.
Uncommon adverse reactions (≥1/100 to <1/10) are syncope.
Contraindications
Hypersensitivity to the active substance or to any of the excipients, or trace residues of tetracycline; active or suspected malignancy; history of malignancies in the gastrointestinal tract including the hepatobiliary system within the last five years.
Special warnings and precautions for use
Colo-rectal polyps; gastrointestinal neoplasia including hepatobiliary tract; gallbladder and bile ducts; pancreatic diseases; monitoring of small bowel, gallbladder and bile ducts, and pancreas; intestinal obstruction; cardiovascular; management of fluids during treatment with Revestive; concomitant medication; special clinical conditions; hepatic impairment; discontinuation of treatment; excipients.
For the current Revestive EU Summary of Product Characteristics, please click here. For the Revestive Product Monograph (Canada), please click here. For the GATTEX U.S. Prescribing Information, please click here.
Shire enables people with life-altering conditions to lead better lives.
Our strategy is to focus on developing and marketing innovative specialty medicines to meet significant unmet patient needs.
We focus on providing treatments in Rare Diseases, Neuroscience, Gastrointestinal and Internal Medicine and we are developing treatments for symptomatic conditions treated by specialist physicians in other targeted therapeutic areas, such as Ophthalmics.
www.shire.com
Date of preparation: May 2016
INTSP/C-ANPROM/TED/16/0036
S12179
iv Carter BA, et al. Clin Nutr 2015;34(Suppl 1):S239.
v Carter BA, et al. J Pediatr Gastroenterol Nutr 2015;61(Suppl 2):S46-47.
vi Wales PW, Christison-Lagay ER. Semin Pediatr Surg 2010; 19(1): 3-9.
vii Sulkowski JP, Minneci PC. Pathophysiology 2014;21(1):111-118.
viii Kaufman SS, Matsumoto CS. Minerva Pediatr 2015;67(4):321-340.
ix Neelis EG, Roskott AM, Dijkstra G, et al. Clin Nutr 2016;35(1):225-229.
SOURCE: Shire
Post Views: 19
