Business
Napo Pharmaceuticals, a Jaguar Health Company, Announces Activation by FDA of Investigational New Drug (IND) Application for Crofelemer for Treatment of Microvillus Inclusion Disease
MVID is a life-threatening and ultra-rare autosomal recessive disease that affects newborns and children, leading to intestinal failure, significant morbidity
About this update from Jaguar Health, Inc.
[{"type":"text","content":"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 diarrheaSAN FRANCISCO, CA / ACCESSWIRE / August 8, 2023 / Napo Pharmaceuticals Inc. (Napo), a Jaguar Health, Inc. (NASDAQ:JAGX) company, today announced the activation by the U.S. Food and Drug Administration (FDA) of the Investigational New Drug (IND) application for a new crofelemer powder for oral solution formulation for the treatment of microvillus inclusion disease (MVID), an ultra-rare congenital diarrheal disorder (CDD).\"We were very pleased to hear that the FDA has communicated that we may proceed with the proposed phase 2 clinical trial for crofelemer for treatment of MVID in pediatric patients with a novel formulation of crofelemer,\" said Lisa Conte, Napo and 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 is supporting investigator-initiated proof-of-concept studies of crofelemer for SBS with intestinal failure and MVID in the EU and Middle East/North Africa (MENA) regions. In accordance with the guidelines of specific EU countries, published d...