Napo Pharmaceuticals Submits Investigational New Drug (IND) Application to FDA
MVID is a life-threatening and ultra-rare autosomal recessive disease that affects newborns and children, leading to intestinal failure, significant morbidity and even death from severe secretory diarrhea
SAN FRANCISCO, CA / ACCESSWIRE / June 28, 2023 / Napo Pharmaceuticals Inc., a Jaguar Health, Inc. (NASDAQ:JAGX) company, today announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a new crofelemer powder for oral solution formulation for the treatment of microvillus inclusion disease (MVID).
"I am very happy that we have submitted the IND for crofelemer powder for oral solution for the treatment of MVID, an ultra-rare congenital diarrheal disorder (CDD)," said Lisa Conte, Jaguar's President and CEO. "MVID is a life-threatening and rare autosomal recessive disease that affects newborns and children, leading to intestinal failure, significant morbidity, and even death from severe secretory diarrhea. While there are currently no approved therapeutic treatments for MVID, parenteral nutrition (PN), the standard of care for management of MVID, can cost up to $150,000 a year or more with complications. MVID patients suffer from severe cholera-like diarrhea, and symptomatic management of diarrhea in MVID may reduce their dependence on PN. We plan to host an investor-facing webinar in the near future with leading pediatric gastroenterologists to further elaborate the value of managing diarrhea in MVID patients with intestinal failure."
Crofelemer has been granted Orphan Drug Designation (ODD) by the FDA and the European Medicines Agency (EMA) for MVID. The ODD program in both the US and European Union qualifies sponsors to receive potential incentives to develop therapies for the diagnosis, prevention, or treatment of rare diseases or conditions. Crofelemer has also been granted ODD by the FDA and the EMA for short bowel syndrome (SBS). SBS patients with intestinal failure are also treated with PN. Jaguar company Napo Pharmaceuticals plans to support investigator-initiated trials of crofelemer for SBS with intestinal failure.