Jaguar Health (NASDAQ:JAGX) is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to crofelemer for the treatment of microvillus inclusion disease (MVID), a rare congenital diarrheal disorder (CDD). Following an extensive review of the submitted application, the FDA has granted this important designation to crofelemer, which can now be used to treat this debilitating condition.
Jaguar’s President and CEO Lisa Conte is thrilled to announce yet another accomplishment for their oral botanical drug, crofelemer. This new molecular entity now holds four orphan designations, having previously received ODD for MVID from the European Medicines Agency (EMA) and for short bowel syndrome (SBS) from both the FDA and the EMA. This is a huge milestone for crofelemer and for Jaguar.
Napo Therapeutics, the Italian corporation founded by Jaguar in 2021, is dedicated to expanding access to crofelemer, a groundbreaking treatment and management solution, across Europe in order to address rare and orphan diseases. Initially focused on CDD and SBS, the Milan-based company is determined to make a real difference in the lives of those affected.
Jaguar is committed to exploring the potential of crofelemer in improving the condition of patients suffering from Short Bowel Syndrome and Congenital Diarrhea with Intestinal Failure. By 2023, proof-of-concept studies will have been conducted, and results published in accordance with European Union guidelines. This could potentially pave the way for early patient access to crofelemer, via European reimbursement programs, and generate much-needed revenue for those affected.
MVID is an incredibly rare and life-threatening disorder that primarily affects newborns and children, causing considerable morbidity and even death due to extreme secretory diarrhea. Fortunately, we now have an understanding of the heterogeneous etiology of CDDs, which are inherited chronic enteropathies, and are able to take a step closer towards discovering treatments for this debilitating disease. According to Pravin Chaturvedi, PhD, Jaguar’s Chair of the Scientific Advisory Board and Chief Scientific Officer, by delving into the different pathophysiologic mechanisms of each type of CDD, we can make progress in finding solutions to help those suffering from MVID.
Patients suffering from both CDD and SBS must grapple with the daily challenges of intestinal failure, often requiring a hefty dose of parenteral nutrition for up to seven days each week. Not only is this associated with a significant risk of morbidity and mortality, it comes with a hefty price tag. The primary symptom of both of these conditions is severe chronic diarrhea, which can lead to serious secondary symptoms such as dehydration, metabolic acidosis or alkalosis, and malnutrition. In some cases, these symptoms can be life-threatening if left untreated.
The Orphan Drug Act (ODA) in the US offers a unique opportunity for small molecule drugs and biological products to be granted special status to treat rare diseases or conditions. Sponsors of the drug can take advantage of various development incentives such as tax credits for qualified clinical testing and filing fee relief. Furthermore, the ODA provides the first sponsor who obtains marketing approval for the designated orphan drug with a seven-year period of marketing exclusivity.
In the EU, obtaining an Orphan Drug Designation (ODD) can be incredibly beneficial for sponsors. Not only do sponsors receive Scientific Advice from the European Medicines Agency (EMA) for clinical trials in the orphan indication, but they also get to enjoy a ten-year market exclusivity when their drug is approved for commercialization. It’s an amazing opportunity for sponsors to get their medicines approved and make an impact in the health of those suffering from rare diseases.
Crofelemer is an exciting breakthrough in the medical world; it is the only oral FDA approved drug under botanical guidance and is derived from the red bark sap of the Croton lechleri tree in the Amazon Rainforest. Developed by Napo Pharmaceuticals, a wholly owned U.S. subsidiary of Jaguar Health, this amazing plant-based drug is sustainably harvested and purified in accordance with fair trade practices, ensuring quality, ecological integrity, and support for Indigenous communities.
About Jaguar Health, Napo Pharmaceuticals, Napo Therapeutics & Jaguar Animal Health
Jaguar Health, Inc. is a commercial stage pharmaceuticals company on a mission to revolutionize the way people and animals with GI distress, including chronic, debilitating diarrhea, are treated. Utilizing proprietary plant-based medicines from responsibly harvested rainforest sources, the company is conducting a pivotal Phase 3 clinical trial (OnTarget study) for the prophylaxis of diarrhea in adult cancer patients receiving targeted therapy. Moreover, Jaguar Health’s wholly owned subsidiary, Napo Pharmaceuticals, Inc., is also expanding crofelemer access in Europe through its Italian corporation Napo Therapeutics S.p.A. (f/k/a Napo EU S.p.A.). As such, Jaguar Health is at the forefront of a new era of sustainable healthcare – the era of Jaguar Animal Health.