Nutrinia, developing therapies to treat rare gastrointestinal disorders, announced today that the U.S. Food and Drug Administration (FDA) granted orphan drug designation for the company's orally-administered drug for treating short bowel syndrome (SBS) in patients of all ages. Nutrinia plans to begin a pivotal clinical trial in this indication. The new treatment is aimed at helping thousands of patients worldwide, reducing their dependence on parenteral nutrition, which puts them at a higher risk for liver disease, infections, and other complications.
SIGNIFICANT MILESTONE ON THE WAY TO TREATING RARE GI DISEASES
"Receiving Orphan Drug Designation in the U.S. market for the development of our SBS therapy, NTRA-9620, to benefit SBS patients represents a significant milestone for Nutrinia," said Miki Olshansky, CEO, Nutrinia. "As a first step, we are working closely with leading neonatologists, gastroenterologists and surgeons, as well as U.S.A FDA to finalize the design of our upcoming trial of NTRA-9620 in infants with SBS."
Subsequent to Nutrinia's Investigational New Drug (IND) application, the company expects to launch a U.S. and European multi-national study in this patient population. The company also plans to run a separate, concurrent clinical program of NTRA-2112 for the treatment of gastrointestinal (GI) malabsorption caused by intestinal immaturity in preterm infants.
"We believe that both of our lead development programs have the potential to safely enhance bowel adaptation and nutrients absorption to improve outcomes for both patient populations," she added.
BETTER TREATMENTS NEEDED
SBS is a malabsorption disorder, frequently caused by the surgical removal of a large portion of the small intestine. Additional causes for SBS in infants are GI-related complications such as necrotizing enterocolitis (NEC), as well as gastroschisis, atresia and other congenital disorders.
The current strategy for treating infants with SBS consists of nutritional, pharmacologic, and surgical interventions. These aim to promote enteral nutrition while minimizing the complications of parenteral nutrition therapy. Many patients must be supported by long-term parenteral treatment, which puts them at risk of liver disease, infections, and other complications. Neonates who have significant SBS frequently suffer from intestinal failure-associated liver disease, catheter-associated bloodstream infections and bacterial overgrowth. Additionally, parenteral nutrition places significant economic burdens on families and the healthcare system, with annual costs ranging from an estimated $180,000 to $570,000.
NUTRINIA'S THERAPIES FOR RARE GI CONDITIONS
The NTRA-9620 drug product is intended to enhance post-resection bowel adaptation and improve patient outcomes. This includes reducing reliance on parenteral nutrition support and increasing enteral nutrition intake.
Both NTRA-9620 and NTRA-2112 are administered orally and target activation within the local GI tract. The active ingredient in Nutrinia's therapeutics is insulin that is specifically formulated to deliver localized treatment of the GI tract without systemic exposure. This route of insulin administration is known to play a key role in supporting the maturation and rehabilitation of the GI tract. Studies have shown that insulin, naturally present in mother's milk, stimulates intestinal growth, cell maturation and enzyme expression. These effects are crucial for normal GI development and better absorption of oral/enteral nutrition. Pre-term infants and patients suffering from intestinal failure are commonly intolerant to the level of enteral nutrition essential to support life, normal growth and development. Enhancing GI functionality and absorption is extremely important in these patient populations.
Nutrinia formulated its products with excipients that are considered very safe and are already approved for infant consumption. Early clinical studies suggest that the therapies increase enteral nutrition absorption, which is associated with improved long-term outcomes.