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Jaguar Health Announces Submission of Orphan Drug Designation Application to the FDA for Crofelemer for Cholera-Related Diarrhea

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Jaguar Health (JAGX) submits an Orphan Drug Designation application to the FDA for crofelemer, a plant-based drug, for the treatment of cholera-related diarrhea. The company aims to address rare diseases with crofelemer and expects results from proof-of-concept studies for microvillus inclusion disease (MVID) and short bowel syndrome (SBS) by 2025. Crofelemer has been granted ODD for MVID and SBS by the FDA and EMA. The potential market exclusivity and development incentives for crofelemer are highlighted, targeting a significant global health issue.
Positive
  • Jaguar Health (JAGX) submits an Orphan Drug Designation application to the FDA for crofelemer for cholera-related diarrhea.
  • The company focuses on rare diseases with crofelemer and expects results from proof-of-concept studies for MVID and SBS by 2025.
  • Crofelemer has been granted ODD for MVID and SBS by the FDA and EMA, providing potential market exclusivity and development incentives.
  • The global health impact of cholera is highlighted, with millions of cases and thousands of deaths reported annually.
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  • None.

The submission of an Orphan Drug Designation (ODD) application for crofelemer by Napo Pharmaceuticals represents a significant strategic move within the pharmaceutical industry. Crofelemer's potential to provide symptomatic relief for moderate-to-severe diarrhea caused by infections, including cholera, positions it as a key player in addressing a global health concern. The rarity of cholera in the U.S. means that the ODD could provide Jaguar Health with substantial benefits, including tax credits, relief from filing fees and a seven-year marketing exclusivity upon approval. This exclusivity is a crucial competitive advantage, potentially protecting the company's market share against generic competition.

From a medical research perspective, the ongoing and expected clinical trials are pivotal. The involvement of reputable institutions like Cleveland Clinic enhances the credibility of the studies. The results from these trials could not only pave the way for FDA and EMA approvals but also set the stage for early patient access in specific European Union countries. The impact of this could be substantial, as early patient access can lead to real-world data that might further support the drug's efficacy and safety profile.

Investors should note the potential financial implications of the ODD application for crofelemer. The designation could streamline the development process and reduce costs through tax credits and fee waivers, which could positively impact Jaguar Health's financials. Additionally, the seven-year U.S. marketing exclusivity and potential ten-year exclusivity in the EU upon approval could safeguard revenue streams for the company, making it an attractive proposition for long-term investment.

However, the market for orphan drugs is typically smaller, given the low incidence of such diseases. Investors should weigh the potential market size against the benefits of exclusivity and consider the broader implications of Jaguar Health's pipeline and the versatility of crofelemer in treating multiple rare indications. The preliminary data from WHO indicating a global resurgence of cholera underscores the relevance of such treatments and suggests a growing market need that could be addressed by crofelemer if approved.

The global health implications of an effective treatment for cholera cannot be overstated. Cholera remains a significant public health threat in many parts of the world, with millions of cases annually leading to tens of thousands of deaths. The potential approval and distribution of crofelemer could have a profound impact on cholera management, particularly in endemic regions. The WHO's classification of the global resurgence of cholera as a grade 3 emergency highlights the urgency for new treatments.

Should crofelemer be approved, it could improve outcomes for patients with severe disease, reducing the rapid dehydration and shock that can lead to death within hours. Moreover, the availability of a plant-based prescription drug could align with global initiatives to find sustainable and accessible treatments for neglected tropical diseases, potentially reshaping the pharmaceutical approach to global health crises.

Cholera is an acute diarrheal illness caused by infection of the intestine with the bacterium Vibrio cholerae

Crofelemer previously granted Orphan Drug Designation by the FDA and the EMA for both short bowel syndrome and microvillus inclusion disease

SAN FRANCISCO, CA / ACCESSWIRE / February 12, 2024 / Jaguar Health (NASDAQ:JAGX) today announced that Jaguar family company Napo Pharmaceuticals (Napo) has submitted an Orphan Drug Designation (ODD) application to the U.S. Food and Drug Administration (FDA) for crofelemer, the company's novel plant-based prescription drug, for the symptomatic relief and treatment of moderate-to-severe diarrhea, with or without concomitant antimicrobial therapy, from bacterial, viral, and parasitic infections including Vibrio cholerae, the bacterium that causes cholera.

Due to the very low incidence and prevalence of cholera in the United States, cholera is an orphan indication in this country. Rare diseases, also referred to as orphan indications, are a core Jaguar development focus. In 2024 and 2025, Jaguar expects results from 5 proof-of-concept studies of crofelemer, the company's novel plant-based prescription drug, from at least 8 clinical sites, including Cleveland Clinic, on three continents for the rare disease indications of microvillus inclusion disease (MVID) - an ultrarare congenital diarrheal disorder (CDD) - and short bowel syndrome (SBS). In accordance with the guidelines of specific European Union countries, published data from such clinical investigations could support reimbursed early patient access to crofelemer for these debilitating conditions in 2025 while the company pursues approval of crofelemer for SBS and MVID from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). In August 2023 the FDA activated Napo's Investigational New Drug (IND) application for a new crofelemer powder for oral solution formulation for the treatment of MVID.

"We are very pleased that the ODD application for crofelemer for cholera-related diarrhea has been submitted," said Steven King, PhD, Jaguar's Chief Sustainable Supply, Ethnobotanical Research & IP Officer. "The company previously presented Phase 2 data on crofelemer for the treatment of devastating dehydration in cholera patients from the renowned International Centre for Diarrhoeal Disease Research (icddr,b) in Bangladesh. Receipt of orphan drug designation would qualify the company for various development incentives, including tax credits for qualified clinical testing and relief of filing fees, and provide the company with a seven-year period of marketing exclusivity if marketing approval is approved for crofelemer for this indication in the U.S. We expect to pursue ODD designation for crofelemer for this indication from the European Medicines Agency (EMA) as well in the future."

Crofelemer has been granted ODD by the FDA and the EMA for both MVID and SBS. The Orphan Drug Act (ODA) in the U.S. provides for granting special status to a small molecule drug or biological product to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan drug designation (or sometimes "orphan status"). In the EU, receipt of ODD supports some specific regulatory pathways, and 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.

Although cholera is an orphan indication in the U.S., it is estimated that, worldwide, 1.3 to 4 million cholera cases and 21,000 to 143,000 cholera-related deaths occur each year, according to the Centers for Disease Control and Prevention of the U.S. Department of Health & Human Services. Approximately one in 10 infected persons will have severe disease characterized by profuse watery diarrhea, vomiting, and leg cramps. In these people, rapid loss of body fluids leads to dehydration and shock. Without treatment, death can occur within hours. Cholera is now endemic in many countries outside the U.S. Preliminary data issued January 11, 2024 by the World Health Organization (WHO) indicates that the number of cholera cases reported in 2023 in WHO Member States as of December 15, 2023 surpassed that of 2022. Nearly a year has passed since WHO classified the global resurgence of cholera as a grade 3 emergency, the highest internal level for a health emergency requiring a comprehensive response at the three levels of the organization.

About Crofelemer

Crofelemer is a botanical (plant-based) drug extracted and purified from the red bark sap, also referred to as "dragon's blood," of the medicinal Croton lechleri tree in the Amazon Rainforest. Jaguar Health family company Napo Pharmaceuticals has established a sustainable harvesting program, under fair trade practices, for crofelemer to ensure a high degree of quality, ecological integrity, and support for Indigenous communities.

About the Jaguar Health Family of Companies

Jaguar Health, Inc. (Jaguar) is a commercial stage pharmaceuticals company focused on developing novel proprietary prescription medicines sustainably derived from plants from rainforest areas for people and animals with gastrointestinal distress, specifically associated with overactive bowel, which includes symptoms such as chronic debilitating diarrhea, urgency, bowel incontinence, and cramping pain. Jaguar family company Napo Pharmaceuticals focuses on developing and commercializing human prescription pharmaceuticals for essential supportive care and management of neglected gastrointestinal symptoms across multiple complicated disease states. Napo Pharmaceuticals' crofelemer drug product candidate is the subject of the OnTarget study, a pivotal Phase 3 clinical trial for preventive treatment of chemotherapy-induced overactive bowel (CIOB) in adults with cancer on targeted therapy. Jaguar family company Napo Therapeutics is an Italian corporation Jaguar established in Milan, Italy in 2021 focused on expanding crofelemer access in Europe and specifically for orphan and/or rare diseases. Jaguar Animal Health is a Jaguar tradename. Magdalena Biosciences, a joint venture formed by Jaguar and Filament Health Corp. that emerged from Jaguar's Entheogen Therapeutics Initiative (ETI), is focused on developing novel prescription medicines derived from plants for mental health indications.

For more information about:

Jaguar Health, visit https://jaguar.health

Napo Pharmaceuticals, visit www.napopharma.com

Napo Therapeutics, visit napotherapeutics.com

Magdalena Biosciences, visit magdalenabiosciences.com

Visit Jaguar on LinkedIn: https://www.linkedin.com/company/jaguar-health/

Visit Jaguar on X: https://twitter.com/Jaguar_Health

Visit Jaguar on Instagram: https://www.instagram.com/jaguarhealthcommunity/

Forward-Looking Statements

Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that results from 5 proof-of-concept studies of crofelemer from at least 8 clinical sites, including Cleveland Clinic, on three continents will be available in 2024 and 2025 for the indications of MVID and SBS, Jaguar's expectation that, in accordance with the guidelines of specific European Union countries, published data from such clinical investigations could support reimbursed early patient access to crofelemer for MVID and SBS in 2025 while the company pursues approval of crofelemer for SBS and MVID from the EMA and the FDA, and Jaguar's expectation that it will pursue ODD designation for crofelemer for cholera-related diarrhea from the EMA in the future. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "aim," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions. The forward-looking statements in this release are only predictions. Jaguar has based these forward-looking statements largely on its current expectations and projections about future events. These forward-looking statements speak only as of the date of this release and are subject to a number of risks, uncertainties and assumptions, some of which cannot be predicted or quantified and some of which are beyond Jaguar's control. Except as required by applicable law, Jaguar does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Source: Jaguar Health, Inc.

Contact:
hello@jaguar.health

SOURCE: Jaguar Health, Inc.



View the original press release on accesswire.com

Jaguar Health submitted an Orphan Drug Designation application for crofelemer, a plant-based drug, for the treatment of cholera-related diarrhea.

Jaguar Health aims to address microvillus inclusion disease (MVID) and short bowel syndrome (SBS) with crofelemer.

Crofelemer has been granted Orphan Drug Designation for MVID and SBS by the FDA and the EMA.

ODD can offer market exclusivity and development incentives for crofelemer, aiding in its commercialization.

Worldwide, 1.3 to 4 million cholera cases and 21,000 to 143,000 cholera-related deaths occur each year.
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About JAGX

jaguar health, inc. is a commercial stage natural-products pharmaceuticals company focused on developing novel, sustainably derived gastrointestinal products for both human prescription use and animals on a global basis. our wholly-owned subsidiary, napo pharmaceuticals, inc., focuses on developing and commercializing proprietary human gastrointestinal pharmaceuticals for the global marketplace from plants used traditionally in rainforest areas. our mytesi® (crofelemer) product is approved by the u.s. fda for the symptomatic relief of noninfectious diarrhea in adults with hiv/aids on antiretroviral therapy. canalevia™ is our lead animal prescription drug candidate, intended for treatment of various forms of diarrhea in dogs. equilevia™ is jaguar’s non-prescription product for total gut health in equine athletes. to read our full social media policy, please visit https://jaguar.health/social-media-policy/