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GATTEX in pediatrics ≥ 1 year

GATTEX® (teduglutide) is the first and only FDA-approved analog of naturally
occurring GLP-2 for patients ≥1 year of age with short bowel syndrome (SBS) who
are dependent on parenteral support (PS)1

Penelope, GATTEX Patient
with her mom, kelsey

GATTEX Mechanism of Action

How GATTEX works

GATTEX amino chain

Half-life of GATTEX in patients with SBS1,2

~42 Minutes

(AGE: 1-11 YEARS)

~60 MInutes

(AGE: 12-17 YEARS)

Half-life of naturally occurring GLP-2 is ~7 minutes1,2

The amino acid sequence of naturally occurring GLP-2 and GATTEX are nearly identical, except for a single amino acid substitution—alanine replaced with glycine.1-4

GLP-2 amino chain

GLP-2=glucagon-like peptide-2

GATTEX (teduglutide) enhanced intestinal absorption in adult patients with short bowel syndrome (SBS)1,3

Villus height after treating with GATTEX

Increased
Villus Height

Increased
Crypt Depth

Not an actual biopsy. Image is for illustrative purposes only and does not represent results from the study.

GATTEX enhanced gastrointestinal fluid (wet weight) absorption in adults by approximately 750–1000 mL/day1

The ability of GATTEX to improve intestinal absorption in children has not been investigated.

The ability of GATTEX (teduglutide) to improve intestinal absorption was studied in 17 adult subjects with SBS (n=2-3 per dose group) using daily doses of 0.03, 0.1, or 0.15 mg/kg (doses ranging from 0.6 to 3 times the recommended dose) in a 21-day, open-label, multicenter, dose-ranging study. Fourteen of 17 patients were dependent on PS. All subcutaneous (abdomen) doses studied, except 0.03 mg/kg once daily, resulted in enhanced gastrointestinal fluid (wet weight) absorption of approximately 750 to 1000 mL/day, and increased villus height and crypt depth of the intestinal mucosa.1

The recommended dosage of GATTEX for both adult and pediatric patients is 0.05 mg/kg once daily by subcutaneous injection. The recommended dosage in adult and pediatric patients with moderate and severe renal impairment and end-stage renal disease (estimated glomerular filtration rate [eGFR] <60 mL/min/1.73 m2) is 0.025 mg/kg once daily.1

Understanding the GATTEX
Mechanism of Action

Share the video below with your patients to see how GATTEX works.

Actors portrayal

Pivotal study of GATTEX (teduglutide) in pediatric patients

In the pivotal Phase 3, 6-month, multicenter, clinical study of GATTEX in pediatric patients aged 1 through 17 years with short bowel syndrome (SBS) who were dependent on parenteral support (PS) (N=59)1,5,6:

  • Caregivers chose for patients to receive either GATTEX (n=50) or standard of care (SOC) (n=9)
    • — Patients in the GATTEX arm were randomized to 0.025 mg/kg/day (n=24) or 0.05 mg/kg/day (n=26)
  • Patients had SBS resulting from major intestinal resection and were dependent on PS that provided ≥30% of caloric and/or fluid/electrolyte needs for ≥3 months prior to screening
  • PS requirements at baseline in the 6-month study:
    • — Stable PS support (defined as the inability to significantly reduce PS support [ie, 10% or less change in PS or advance in feeds]) for ≥3 months prior to and during screening as assessed by the investigator
    • — PS and specialized enteral nutrition were recorded in intake diaries by the child’s parent or guardian
  • The primary endpoint was a reduction in PS volume of ≥20% from baseline to the end of treatment. Additional endpoints included reduction in PS volume from baseline and infusion hours/day, mean change in baseline PS infusion days/week at Week 24, and enteral autonomy

6-month study1,5,6

Study design

Screening period

Caregivers chose for patients to receive either GATTEX (n=50) or SOC (n=9)

  • Patients in the GATTEX arm were randomized to 0.025 mg/kg/day (n=24) or 0.05 mg/kg/day (n=26)

Enrolled and completed
study: 6 months

GATTEX + SOC

n=24
0.025 mg/kg/day

GATTEX + SOC

n=26
0.05 mg/kg/day

SOC only

n=9

Follow-up period:
4 weeks

SOC only

Patient Baseline Characteristics

INTENT-TO-TREAT POPULATION1,5 GATTEX 0.05 mg/kg/day
(n=26)
DEMOGRAPHICS
Mean age, years (standard deviation [SD]) 6 (4)
1-11 years (%) 24 (92)
12-16 years (%) 2 (8)
17-<18 years (%) 0
Male, n (%) 19 (73)
PRIMARY CAUSES OF SBS, n (%)
Gastroschisis 14 (84)
Midgut volvulus 6 (23)
Necrotizing enterocolitis 3 (12)
Intestinal atresia 1 (4)
Hirschsprung’s disease 1 (4)
Other 1 (4)
BASELINE CHARACTERISTICS
Mean remaining small intestine length, cm (SD) 47 (28)
Stoma, n (%) Jejunostomy 5 (18)
4 (80)
Patients with remaining colon, n (%) Colon-In-continuity 25 (96)
22 (88)
Mean actual PS volume, mL/kg/day (SD) 60 (29)
Mean PS infusion time, days/week (SD) 7 (1)
Mean PS infusion time, hours/day (SD) 11 (3)

See how to get your appropriate patients started, or keep reading to explore results with GATTEX, safety profile, and dosing.

As Penelope's mother, watching us reduce days of PS to get us where we are now has been the most rewarding experience in our SBS journey to date. She now sleeps line-free, and we have a lot more time together as a family.”

Kelsey,
Mother of Gattex Patient

GATTEX reduced weekly parenteral support (PS) volume for the majority of pediatric patients1,5

Reduction in PS volume requirements1,5

Percentage of patients who achieved ≥20% reduction in weekly parenteral support (PS) volume from baseline

At 6 months:

69%
(18/26)
of patients treated with GATTEX achieved ≥20% reduction in weekly PS volume1,5‡§¶

GATTEX reduced mean weekly parenteral support (PS) volume over time1,5‖

PS volume requirements were reduced by 23 mL/kg/day (±18), which was a 42% (±29) reduction from baseline§

VISIT ( W EE K S) 1 15 4 18 7 2 1 10 2 16 5 19 8 22 11 3 1 7 6 20 9 2 3 12 13 14 24 G A TTEX 0 . 05 mg/ k g / d a y (n=26) M E AN PE R C EN T A G E CH AN G E F R OM B A SELI N E IN PS V OLUME (mL/ k g / d a y) - 2 3 mL / k g / d a y 1 8 ) - 4 2 % 2 9 % ) 0 -50 -40 -30 -20 -10

Actors portrayal

GATTEX reduced the amount of time pediatric patients spent on parenteral support (PS)1,5

Time off of PS1,5

Patients who achieved time off of PS

At 6 months¶‖:

Patients treated with GATTEX achieved a mean reduction in time of
3
HOURS/DAY off of PS5#
(±3.8 Hours/Day)
38%
(10/26)
patients treated with GATTEX spent
≥1 fewer days per week on PS1,5**
Primary efficacy endpoint.
Results based on patient diary data from the intent-to-treat population.
§Weight-normalized reduction.
GATTEX 0.05 mg/kg/day dosage group.
Key secondary efficacy endpoint.
#11 hours/day mean baseline PS infusion time.
**7 days/week mean PS infusion time.

GATTEX helped some pediatric patients achieve complete freedom from parenteral support (PS)1,5

Complete freedom from PS

Number of patients who achieved complete freedom from PS††‡‡

At 6 months:

3
Patients
(3/26, 12%)
patients treated with GATTEX were able to wean off PS completely1,5
††GATTEX 0.05 mg/kg/day dosage group.
‡‡Key secondary efficacy endpoint.

GATTEX has a demonstrated safety profile1

In 2 clinical studies, 41 pediatric patients were treated with GATTEX 0.05 mg/kg/day1

  • Overall, the safety profile of GATTEX for pediatric patients aged 1 to <17 years was similar to that for adults1
  • The 41 patients included 1 infant (1 to <2 years), 37 children (2 to <12 years), and 3 adolescents (12 to <17 years)1
  • In the long-term extension studies with a mean exposure of 41 weeks, no new safety signals were identified1
MOST COMMON ADVERSE REACTIONS IN ADULTS1
(≥5% in the GATTEX group and greater than in the placebo group)
Placebo
(n=59)
(%)
GATTEX 0.05 mg/kg/day
(n=77)
(%)
Abdominal pain§§ 22 30
Nausea 20 23
Upper respiratory tract infection¶¶ 12 21
Abdominal distension 2 20
Injection site reaction|| || 12 13
Vomiting 10 12
Fluid overload## 7 12
Hypersensitivity*** 7 10
Flatulence 7 9
Decreased appetite 3 7
Influenza†† 2 7
Skin hemorrhage‡‡‡ 2 5
Cough 0 5
Sleep disturbances§§§ 0 5
  • If pediatric patients have a stoma, advise patients and caregivers that, while they may experience abdominal pain and swelling of their stoma, especially when starting treatment with GATTEX, if they experience symptoms of intestinal obstruction, they should contact their physician1

Among the 53 patients with a stoma in the placebo-controlled studies, the percentage of patients with gastrointestinal stoma complication was 42% (13/31) for patients who were treated with GATTEX 0.05 mg/kg/day and 14% (3/22) for patients who received placebo.1

§§ Includes: Abdominal pain, upper abdominal pain, lower abdominal pain.

¶¶ Includes: Upper respiratory tract infection, nasopharyngitis, pharyngitis, sinusitis, laryngitis, rhinitis, viral upper respiratory tract infection.

|| || Includes: Injection site hematoma, injection site erythema, injection site pain, injection site swelling, injection site hemorrhage, injection site discoloration, injection site reaction, injection site rash.

## Includes: Fluid overload, peripheral edema, edema, generalized edema, fluid retention, and jugular vein distension.

*** Includes: Erythema, rash, dermatitis allergic, pruritus, rash macular, drug eruption, eyelid edema, flushing.

†† Includes: Influenza, influenza-like illness.

‡‡‡ Includes: Hematoma, abdominal wall hematoma, post-procedural hematoma, umbilical hematoma, blood blister.

§§§ Includes: Insomnia (3 patients) and hypersomnia (1 patient).

Monitoring timeline for pediatric patients receiving GATTEX

Within 6 months prior to starting GATTEX treatment1:

  • Perform fecal occult blood testing; if there is unexplained blood in the stool, perform colonoscopy/sigmoidoscopy1
  • Obtain baseline laboratory assessments (bilirubin, alkaline phosphatase, lipase, and amylase)1
Ongoing Every 6
months
After 1 year
of GATTEX¶¶¶
Every
year
At least every
5 years
Fecal occult
blood test1
Examine for unexplained blood in stool
Colonoscopy/
Sigmoidoscopy1¶¶¶
Assess for colorectal polyps
Laboratory assessments1 Bilirubin
Alkaline phosphatase
Lipase
Amylase
Clinical
evaluations1
Signs and symptoms of intestinal obstruction
Signs and symptoms of fluid imbalance and fluid overload
Increased absorption of concomitant oral medication(s)
Observation of other adverse events
Fecal occult blood test every year1
Examine for unexplained blood in stool
Colonoscopy/Sigmoidoscopy after 1 year of GATTEX and every 5 years thereafter 1¶¶¶
Assess for colorectal polyps
Laboratory assessments every 6 months1
Bilirubin
Alkaline phosphatase
Lipase
Amylase
Ongoing Clinical Evaluations1
Signs and symptoms of intestinal obstruction
Signs and symptoms of fluid imbalance and fluid overload
Increased absorption of concomitant oral medication(s)
Observation of other adverse events

Discontinuation of treatment with GATTEX may result in fluid and electrolyte imbalance. Fluid and electrolyte status should be monitored in patients who discontinue treatment with GATTEX.1

¶¶¶Colonoscopy/sigmoidoscopy is recommended for all children and adolescents after 1 year of treatment, every 5 years thereafter while on continuous treatment with GATTEX, and if they have new or unexplained gastrointestinal bleeding.1

See how to get your appropriate patients started, or keep reading for information on dosing.

GATTEX Pediatric
Dosing & Administration1

The recommended dosage in pediatric patients 1 year of age and older is 0.05 mg/kg once daily by subcutaneous injection

  • The recommended dosage in pediatric patients with moderate and severe renal impairment and end-stage renal disease (estimated glomerular filtration rate less than 60 mL/min/1.73 m2) is 0.025 mg/kg once daily
  • GATTEX is available in a 5 mg vial
  • Use of the GATTEX 5 mg kit is not recommended in pediatric patients weighing less than 10 kg

Calculation of weight-based dosing

patient weight (kg)

patient weight (kg)

Multiply by 0.05 OR
0.025 per above

Divide by 200 (0.05 dose)
OR 400 (0.025 dose)

patient dose (mg/day)

volume (mL/day)

GATTEX should always be administered by a caregiver

  • Full preparation and injection instructions can be found in the GATTEX Instructions for Use
  • Self-administration in pediatric patients has not been tested
  • Caregivers should inject GATTEX into 1 of the 4 quadrants of the abdomen, either thigh, or either arm. They should use a different injection site each time
  • GATTEX should be administered by subcutaneous injection once daily
    — Not for intravenous (IV) or intramuscular (IM) injection
  • GATTEX should be administered within 3 hours after reconstitution

Getting Started

Steps to getting your appropriate patients started on GATTEX

Learn More

  1. GATTEX (teduglutide) for injection [package insert]. Lexington, MA: Shire-NPS Pharmaceuticals, Inc.
  2. Tappenden KA et al. J Clin Gastroenterol. 2013;47(7):602-607.
  3. Jeppesen PB et al. Gut. 2005;54(9):1224-1231
  4. .
  5. Tavares W et al. Am J Physiol Endocrinol Metab. 2000;278(1):E134-E139.
  6. Kocoshis SA et al. JPEN J Parenter Enteral Nutr. 2019. doi:10.1002/jpen.1690.
  7. A 24-week, double-blind, safety, efficacy, and pharmacodynamic study investigating two doses of teduglutide in pediatric subjects through 17 years of age with short bowel syndrome who are dependent on parenteral support. ClinicalTrials.gov identifier: NCT02682381. Accessed May 8, 2020. https://clinicaltrials.gov/ct2/show/study/NCT02682381.
Close Minus Plus
INDICATION

GATTEX® (teduglutide) for injection is indicated for the treatment of adults and pediatric patients 1 year of age and older with short bowel syndrome (SBS) who are dependent on parenteral support.

IMPORTANT SAFETY INFORMATION

Warnings and Precautions
Gattex has been associated with acceleration of neoplastic growth, intestinal obstruction, billary and pancreatic disease, fluid imbalance and fluid overload, and increased absorption of concomitant oral medication. Click here for additional Safety Information.

INDICATION
GATTEX® (teduglutide) for injection is indicated for the treatment of adults and pediatric patients 1 year of age and older with short bowel syndrome (SBS) who are dependent on parenteral support.

IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Acceleration of neoplastic growth
Colorectal polyps were identified during clinical trials. There is a risk for acceleration of neoplastic growth. In adults, within 6 months prior to starting treatment with GATTEX, colonoscopy of the entire colon with removal of polyps should be performed and follow-up colonoscopy (or alternate imaging) is recommended at the end of 1 year of GATTEX. Subsequent colonoscopies should be performed every 5 years or more often as needed.

In children and adolescents, perform fecal occult blood testing prior to initiating treatment with GATTEX. Colonoscopy/sigmoidoscopy is required if there is unexplained blood in the stool. Perform subsequent fecal occult blood testing annually in children and adolescents while they are receiving GATTEX. Colonoscopy/sigmoidoscopy is recommended for all children and adolescents after 1 year of treatment, every 5 years thereafter while on continuous treatment with GATTEX, and if they have new or unexplained gastrointestinal bleeding.

In case of intestinal malignancy (GI tract, hepatobiliary, pancreatic), discontinue GATTEX. The clinical decision to continue GATTEX in patients with non-gastrointestinal malignancy should be made based on benefit-risk considerations.

Intestinal obstruction
Intestinal obstruction has been reported in clinical trials and postmarketing. In patients who develop intestinal or stomal obstruction, GATTEX should be temporarily discontinued pending further clinical evaluation and management.

Biliary and pancreatic disease
Cholecystitis, cholangitis, cholelithiasis, and pancreatitis have been reported in clinical trials and postmarketing. Laboratory assessment (bilirubin, alkaline phosphatase, lipase, amylase) should be obtained within 6 months prior to starting GATTEX. Subsequent laboratory tests should be done every 6 months or more often as needed. If clinically meaningful changes are seen, further evaluation is recommended including imaging, and continued treatment with GATTEX should be reassessed.

Fluid imbalance and fluid overload
Fluid overload and congestive heart failure have been observed in clinical trials. If fluid overload occurs, especially in patients with underlying cardiovascular disease, parenteral support should be adjusted and GATTEX treatment reassessed. If significant cardiac deterioration develops while on GATTEX, continued GATTEX treatment should be reassessed.

Discontinuation of treatment with GATTEX may also result in fluid and electrolyte imbalance. Fluid and electrolyte status should be monitored in patients who discontinue treatment with GATTEX.

Increased absorption of concomitant oral medication
In clinical trials, one patient receiving prazepam concomitantly with GATTEX experienced dramatic deterioration in mental status progressing to coma during first week of GATTEX therapy. Patients receiving concomitant oral drugs requiring titration or with a narrow therapeutic index should be monitored for adverse reactions due to potential increased absorption of the concomitant drug. The concomitant drug may require a reduction in dosage.

Adverse Reactions
The most common adverse reactions (≥ 10%) with GATTEX are abdominal pain, nausea, upper respiratory tract infection, abdominal distension, injection site reaction, vomiting, fluid overload, and hypersensitivity.

Use in Specific Populations
Breastfeeding is not recommended during treatment with GATTEX.

Please click here for full Prescribing Information or Información de prescripción en español.