The data demonstrate that patients who continued Gattex treatment beyond two years achieved clinically meaningful reductions in parenteral support requirements, including volume and days off, with 13 out of 88 patients across all groups achieving complete independence.
In the U.S., Gattex is indicated for the treatment of adult patients with SBS who are dependent on parenteral support. Full prescribing information for Gattex is available by clicking here or visiting www.Gattex.com.
"We are pleased that the FDA has approved updated labeling for Gattex, as it provides important information for healthcare professionals and patients about long-term use of Gattex therapy," said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharma.
"The STEPS 2 study demonstrated that there was an increased response to treatment over time in all groups receiving Gattex in terms of volume reductions, days off and complete weaning of parenteral support."
The efficacy, safety, and tolerability of Gattex was evaluated in a randomized, double-blind, placebo-controlled, parallel-group, multi-national, multi-center clinical trial, known as STEPS, in adults with SBS who were dependent on parenteral nutrition/intravenous (PN/I.V.) support for at least 12 months and required PN at least 3 times per week.
STEPS 2 was a 2-year open-label extension of STEPS in which 88 subjects received Gattex 0.05 mg/kg/day. Ninety-seven percent (76/78) of subjects who completed STEPS elected to enroll in STEPS 2 (37 received Gattex; 39 received Placebo). An additional 12 subjects entered STEPS 2, who had been optimized and stabilized but not randomized in STEPS because of closed enrollment.
Thirty Gattex subjects completed a total duration of 30 months (STEPS followed by STEPS 2 treatment). Of these, 28 subjects (93%) achieved a 20% or greater reduction of parenteral support. Of responders in STEPS who had completed 2 additional years of continuous treatment with Gattex, 96% (21/22) sustained their response to Gattex.
The mean reduction in PN/I.V. (n=30) was 7.55 L/week (a 65.6% reduction from baseline). Ten subjects were weaned off their PN/I.V. support while on Gattex treatment for 30 months. Subjects were maintained on Gattex even if no longer requiring PN/I.V. support. These 10 subjects had required PN/I.V. support for 1.2 to 15.5 years, and prior to Gattex had required between 3.5 L/week and 13.4 L/week of PN/I.V. support. At the end of study, 21 (70%), 18 (60%) and 18 (60%) of the 30 completers achieved a reduction of 1, 2, or 3 days per week in PN/I.V. support, respectively.
Of the 39 placebo subjects from STEPS entering STEPS 2, 29 completed 24 months of treatment with Gattex. The mean reduction in PN/I.V. was 3.11 L/week (an additional 28.3% reduction) from the start of STEPS 2. Sixteen (55.2%) of the 29 completers achieved a 20% or greater reduction of parenteral support.
At the end of study, 14 (48.3%), 7 (24.1%) and 5 (17.2%) achieved a reduction of 1, 2, or 3 days per week in PN/I.V. support, respectively. Two subjects were weaned off their PN/I.V. support while on Gattex. Of the 12 subjects entering STEPS 2 directly, 6 completed 24 months of treatment with Gattex. Similar effects were seen. One of the six subjects was weaned off their PN/I.V. support while on Gattex.
The rates of adverse events of special interest as described in the U.S. Prescribing Information remain consistent. The most common adverse events in STEPS 2 were abdominal pain, catheter sepsis, and episodes of decreased weight, nausea, gastrointestinal stoma complications, and abdominal distension.