NPS Pharmaceuticals, a specialty pharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, announced today its support for the second annual U.S. Rare Disease Day observance on February 28. Rare Disease Day aims to call attention to the 7,000 rare diseases affecting people throughout the world, including nearly 30 million Americans.
“The quality of my life would improve substantially if a new treatment could allow me to avoid my PN-infusions for even one night a week.”
“Patients with rare diseases are a medically underserved population in every country,” said Francois Nader, M.D., president and chief executive officer of NPS Pharmaceuticals. “At NPS, we have dedicated ourselves to developing drugs that target the underlying cause of rare diseases. We are currently focused on two serious orphan conditions: short bowel syndrome (SBS) and hypoparathyroidism, and are developing GATTEX® (teduglutide) and NPSP558, respectively, to address the serious unmet needs of patients with these rare disorders. Patients with both of these conditions face significant challenges and current palliative treatments are associated with serious co-morbidities.”
SBS typically arises after a significant bowel resection, usually due to Crohn’s disease or cancer. Without enough bowel to naturally support their nutritional needs, these patients are left dependent on infusions of parenteral nutrition (PN), the cost of which can exceed $100,000 annually per patient. PN use is associated with potentially life-threatening complications including sepsis and liver damage, and reduced quality-of-life due to the time required for and consequences of frequent access to an intravenous pump.
“Because of my need for infusions, I never get a full night’s sleep. It’s been like caring for a newborn baby for over 28 years without a break,” said Tom Sanford, a Vietnam-era veteran who underwent two bowel resections because of Crohn’s disease in the early 1980s. With only 18-inches of small bowel left, Sanford, a soil conservationist from upstate New York, has been dependent on nightly infusions of PN ever since his last surgery in 1982. “The quality of my life would improve substantially if a new treatment could allow me to avoid my PN-infusions for even one night a week.”
NPS is currently enrolling patients in a Phase 3 study of GATTEX (teduglutide) known as STEPS. STEPS is an international, double-blind, placebo-controlled safety and efficacy study to confirm previously-reported data that showed GATTEX was well tolerated and had a beneficial impact on reduced PN dependence in short-bowl syndrome patients.
The company is also currently enrolling hypoparathyroidism patients into REPLACE, a double-blind, placebo-controlled Phase 3 registration study aimed at demonstrating that once-daily subcutaneous dosing with NPSP558 over a period of 24 weeks is a safe and effective treatment for patients with this rare condition.