jaguar Health, Inc. (JAGX) 0.1407-0.0055 (-3.76%)
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MVID, a rare congenital diarrheal disorder (CDD) condition, is a life-threatening autosomal recessive disease that affects newborns and children and leads to significant morbidity and mortality from intestinal failure, including severe secretory diarrhea
Presentation expected in December 2022 of initial results of an investigator-initiated proof-of-concept trial of crofelemer for CDD and short bowel syndrome (SBS) with intestinal failure, supporting the potential for expanded patient access to crofelemer in 2023 in Europe
Pravin Chaturvedi, PhD, Jaguar's Chief Scientific Officer and Chair of the Scientific Advisory Board, Receives First Boston Biotechnology Summit Lifetime Achievement Award
SAN FRANCISCO, CA / ACCESSWIRE / October 17, 2022 / Jaguar Health (NASDAQ:JAGX) and Napo Therapeutics, the Italian corporation established by Jaguar Health in Italy in 2021 that focuses on development and commercialization of crofelemer in orphan and rare diseases, thus expanding access to crofelemer to patients in Europe, today announced that the European Commission has adopted the decision to grant Orphan Drug Designation (ODD) for crofelemer for the indication of microvillus inclusion disease (MVID), a rare congenital diarrheal disorder (CDD) condition, in the European Union following review of the ODD application Napo Therapeutics submitted to the European Medicines Agency (EMA) in May 2022.
"This is a very welcome development for crofelemer, a new molecular entity that has been granted two orphan designations by the EMA in less than one year, as crofelemer received ODD for short bowel syndrome (SBS) from the EMA in December 2021," said Massimo Mineo, CEO of Napo Therapeutics. "The recognition of ODD in Europe for crofelemer for MVID is a key Napo Therapeutics milestone under the company's exclusive crofelemer license agreement with Jaguar, and receipt of this new ODD supports some specific regulatory pathways for this serious form of CDD, which remains a significant unmet medical need, especially in pediatric patients."
"CDDs are a group of inherited chronic enteropathies characterized by heterogeneous etiology, and each type of CDD is thus a different disease with a different pathogenetic mechanism," said Martire Particco, MD, Chief Medical Officer of Napo Therapeutics. "MVID is a life-threatening and rare autosomal recessive disease that affects newborns and children and leads to significant morbidity and even death from severe secretory diarrhea."
CDD and SBS patients are both subject to intestinal failure, often requiring parenteral nutrition up to 7 days a week, and the immense associated morbidity, mortality, and medical expense. CDD and SBS patients also share a primary common symptom: chronic diarrhea, and therefore associated sequelae from diarrhea, including significant dehydration, metabolic acidosis or alkalosis and malnutrition, and other secondary symptoms, and these symptoms emerge either early or late, and many times become life-threatening.
Napo Therapeutics' mission is to provide access to Jaguar Health's proprietary first-in-class plant-based medicine crofelemer in Europe to address such significant rare disease indications. The company's initial focus is on the clinical development and registration in Europe of crofelemer for short bowel syndrome with intestinal failure (SBS-IF) and subsequently for CDD.
Jaguar is currently supporting an investigator-initiated proof-of-concept study of crofelemer in patients with SBS or CDD with intestinal failure, with a planned endpoint of reduction of requirement of weekly volume of parenteral nutrition. The investigator is targeting a presentation in December 2022 of initial results from the study at a global GI conference in Dubai. In accordance with the guidelines of specific EU countries, publications of such data could support early patient access of crofelemer for SBS or CDD with intestinal failure by mid-2023 through programs in Europe for these devastating diseases for which there is a significant unmet medical need.
Sponsors who obtain ODD for their drug can benefit from Scientific Advice from the EMA for clinical trials for the orphan indication and receive market exclusivity for a period of ten years once the medicine is approved for commercialization.
On October 14, 2022, Pravin Chaturvedi, PhD, Jaguar's Chief Scientific Officer and Chair of the Scientific Advisory Board, received a First Boston Biotechnology Summit Lifetime Achievement Award during the Boston Biotechnology Summit. "We congratulate Dr. Chaturvedi on this wonderful and very well-deserved accomplishment! During his impressive 30+ year career in the pharmaceutical industry, Dr. Chaturvedi has developed and brought to market seven pharmaceutical products in the U.S., and his list of accomplishments includes designing the successful Phase 3 ADVENT trial of crofelemer that resulted in approval of the drug in the U.S., now marketed under the name Mytesi®, for the symptomatic relief of noninfectious diarrhea in adult patients with HIV/AIDS on antiretroviral therapy," said Lisa Conte, Jaguar's president and CEO.
The Boston Biotechnology Summit is an exclusive, Trans-Atlantic bridge designed to foster innovative synergies between biotech and pharma companies, healthcare-focused cities, regional clusters and institutional, philanthropic and strategic investors. The Summit's intent is to spark projects, their financing, and strategic deals to solve unmet medical needs to improve patient lives globally.
