NPS submits NDA CMC section to FDA for GATTEX

NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a specialty pharmaceutical company developing orphan therapeutics for rare gastrointestinal and endocrine disorders, today announced that it has submitted the chemistry, manufacturing and controls (CMC) section of its new drug application (NDA) for GATTEX® (teduglutide) to the U.S. Food and Drug Administration (FDA). GATTEX is a novel, recombinant analog of human glucagon-like peptide 2 that the company is developing for the treatment of adults with short bowel syndrome or SBS. SBS is a highly disabling disorder in which the body is unable to absorb sufficient nutrients and/or fluids through the gastrointestinal tract. Patients with SBS often rely on parenteral nutrition (PN) or intravenous (IV) fluids to survive.

“We believe GATTEX represents an important treatment advance for patients with short bowel syndrome, and we look forward to submitting the remainder of our U.S. marketing application later this year.”

"This rolling submission is an important milestone for NPS and brings us one step closer to commercializing GATTEX," said Francois Nader, MD, president and chief executive officer of NPS Pharmaceuticals. "We believe GATTEX represents an important treatment advance for patients with short bowel syndrome, and we look forward to submitting the remainder of our U.S. marketing application later this year."

FDA regulations permit an applicant to submit the CMC section 90 to 120 days before the anticipated submission of the remainder of the application. FDA will review the early CMC submission as resources permit.

Earlier this year, NPS reported positive data from a 24-week Phase 3 pivotal study of GATTEX. The study, known as STEPS, evaluated the ability of GATTEX to reduce PN and IV fluid requirements of adult subjects with SBS. The primary efficacy endpoint was defined as the percentage of patients who achieved a 20 to 100 percent reduction in weekly PN/IV volume at Weeks 20 and 24, compared to baseline. In the intent-to-treat population, 63 percent (27/43) of GATTEX-treated patients were responders versus 30 percent (13 of 43) of placebo-treated patients.

Subjects treated with GATTEX for 24 weeks achieved significantly greater reductions in weekly PN and IV fluid volume and infusion days versus placebo. At Week 24, subjects who received GATTEX experienced an average 4.4 liter reduction in weekly PN/IV volume from a pre-treatment baseline of 12.9 liters; subjects who received placebo experienced an average 2.3 liter reduction from a pre-treatment baseline of 13.2 liters (p≤0.001). After completing 24 weeks of treatment, 54 percent (21 of 39) of GATTEX-treated subjects were able to reduce the number of infusion days per week by one or more days, compared to 23 percent (9 of 39) of those treated with placebo.

The company is also advancing STEPS 2, an open-label continuation study of STEPS in which each subject receives GATTEX. At an interim update in May 2011, NPS reported that three subjects participating in STEPS 2 were able to gain independence from and discontinue PN and IV fluids.