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FDA Grants Orphan Drug Designation to Jaguar Health for Crofelemer for Microvillus Inclusion Disease (MVID), a Second Rare Disease Indication in the US
Crofelemer was previously granted Orphan Drug Designation by the FDA for short bowel syndromeSAN FRANCISCO, CA / ACCESSWIRE / February 28, 2023 / Jaguar
About this update from Jaguar Health, Inc.
[{"type":"text","content":"Crofelemer was previously granted Orphan Drug Designation by the FDA for short bowel syndromeSAN FRANCISCO, CA / ACCESSWIRE / February 28, 2023 / Jaguar Health (NASDAQ:JAGX) (\"Jaguar\" or the \"Company\") today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to crofelemer for the indication of microvillus inclusion disease (MVID), a rare congenital diarrheal disorder (CDD) condition, following review of the ODD application the Company submitted to the FDA.\"This is another very welcome development for our oral botanical drug, crofelemer, a new molecular entity that now holds four orphan designations, as crofelemer previously received ODD for MVID from the European Medicines Agency (EMA) and for short bowel syndrome (SBS) from both the FDA and the EMA,\" said Lisa Conte, Jaguar's president and CEO.CDD and SBS are the initial focus of Napo Therapeutics, the Italian corporation established by Jaguar in Milan, Italy in 2021 with a mission to expand crofelemer access in Europe for the treatment and management of orphan and rare disease indications.Jaguar is currently supporting investigator-initiated proof-of-concept studies of crofelemer in patients with SBS or CDD with intestinal failure, focused on obtaining proof-of-concept (POC) of reduction of requirements of parenteral support including parenteral nutrition and/or intravenous fluids, throughout 2023. In accordance with the guidelines of specific European Union countries, publications of POC data from these trials could support early patient access to crofelemer for SBS or CDD with intestinal failure in 2023 through programs in Europe. Early access programs are revenue generating, and reimbursable for participating 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 pathophysiologic mechanism,\" said Pravin Chaturvedi, PhD, Jaguar's Chair of the Scientific Advisory Board and Chief Scientific Officer. \"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 subject to intestinal failure, often requiring parenteral nutrition (PN) up to 7 days a week. These condition...