FDA Approves Orphan-Drug Designation for Jaguar Health’s Crofelemer for Treatment of Diarrhea in Cholera
Jaguar Health (NASDAQ:JAGX) announced that the FDA has granted orphan-drug designation (ODD) to crofelemer for treating diarrhea in cholera patients. This plant-based prescription drug previously received Phase 2 data from clinical trials in Bangladesh. The ODD provides development incentives including tax credits and a seven-year marketing exclusivity period if approved.
Crofelemer is currently involved in five rare disease clinical trials, with patient dosing expected throughout December 2024 and Q1 2025. Results could be available by Q2 2025. The company also plans to pursue ODD for a second-generation agent, NP-300, along with a Tropical Disease Priority Review Voucher, which historically has sold for $67-350 million.
Cholera affects 1.3-4 million cases globally annually, with 21,000-143,000 deaths. WHO has classified the global cholera resurgence as a grade 3 emergency, its highest internal level.
Jaguar Health (NASDAQ:JAGX) ha annunciato che la FDA ha concesso la designazione di farmaco orfano (ODD) per il crofelem per il trattamento della diarrea nei pazienti affetti da colera. Questo farmaco prescrittivo a base vegetale ha precedentemente ricevuto dati della Fase 2 da studi clinici in Bangladesh. L'ODD offre incentivi allo sviluppo, tra cui crediti d'imposta e un periodo di esclusività sul mercato di sette anni, se approvato.
Il crofelem è attualmente coinvolto in cinque studi clinici su malattie rare, con somministrazione ai pazienti prevista per tutto dicembre 2024 e il primo trimestre del 2025. I risultati potrebbero essere disponibili entro il secondo trimestre del 2025. L'azienda ha anche in programma di perseguire l'ODD per un agente di seconda generazione, NP-300, insieme a un Voucher di Revisione Prioritaria per malattie tropicali, che storicamente è stato venduto per 67-350 milioni di dollari.
Il colera colpisce annualmente da 1,3 a 4 milioni di casi a livello globale, con 21.000-143.000 decessi. L'OMS ha classificato il ripristino globale del colera come un'emergenza di grado 3, il suo livello interno più alto.
Jaguar Health (NASDAQ:JAGX) anunció que la FDA ha otorgado la designación de medicamento huérfano (ODD) a crofelem para tratar la diarrea en pacientes con cólera. Este medicamento recetado a base de plantas recibió previamente datos de Fase 2 de ensayos clínicos en Bangladesh. La ODD proporciona incentivos para el desarrollo, incluidos créditos fiscales y un período de exclusividad de comercialización de siete años si se aprueba.
Crofelem actualmente está involucrado en cinco ensayos clínicos de enfermedades raras, con dosificación para pacientes prevista durante diciembre de 2024 y el primer trimestre de 2025. Los resultados podrían estar disponibles para el segundo trimestre de 2025. La empresa también planea solicitar la ODD para un agente de segunda generación, NP-300, junto con un Voucher de Revisión Prioritaria para Enfermedades Tropicales, que históricamente se ha vendido por 67-350 millones de dólares.
El cólera afecta entre 1.3 y 4 millones de casos anualmente a nivel global, con 21,000 a 143,000 muertes. La OMS ha clasificado el resurgimiento global del cólera como una emergencia de grado 3, su nivel interno más alto.
재규어 헬스 (NASDAQ:JAGX)는 FDA가 콜레라 환자의 설사를 치료하기 위해 크로펠레머에 고아약 지정(ODD)을 부여했다고 발표했습니다. 이 식물 기반 처방약은 이전에 방글라데시에서 실시된 임상 시험의 2상 데이터를 받았습니다. ODD는 개발 인센티브를 제공하며, 승인될 경우 세금 공제와 7년의 마케팅 독점 기간을 포함합니다.
크로펠레머는 현재 다섯 개의 희귀병 임상 시험에 참여하고 있으며, 환자 투여는 2024년 12월과 2025년 1분기 동안 예정되어 있습니다. 결과는 2025년 2분기까지 제공될 수 있습니다. 이 회사는 또한 2세대 약물 NP-300에 대한 ODD를 추구할 계획이며, 역사적으로 6,700만 달러에서 3억 5천만 달러에 판매된 열대병 우선 검토 바우처도 함께 추진할 예정입니다.
콜레라는 전 세계적으로 매년 130만 건에서 400만 건의 사례가 발생하며, 21,000명에서 143,000명 사망에 이릅니다. WHO는 전 세계 콜레라 재발을 3등급 비상사태로 분류했으며, 이는 그 내부 수준 중 가장 높은 등급입니다.
Jaguar Health (NASDAQ:JAGX) a annoncé que la FDA a accordé la désignation de médicament orphelin (ODD) au crofelem pour le traitement de la diarrhée chez les patients atteints de choléra. Ce médicament sur ordonnance à base de plantes a précédemment reçu des données de Phase 2 d'essais cliniques au Bangladesh. L'ODD offre des incitations au développement, notamment des crédits d'impôt et une période d'exclusivité sur le marché de sept ans si approuvé.
Le crofelem est actuellement impliqué dans cinq essais cliniques sur des maladies rares, avec une administration aux patients prévue tout au long de décembre 2024 et au premier trimestre 2025. Les résultats pourraient être disponibles d'ici le deuxième trimestre 2025. L'entreprise prévoit également de demander l'ODD pour un agent de deuxième génération, NP-300, ainsi qu'un Bon de Révision Prioritaire pour les Maladies Tropicales, qui s'est historiquement vendu entre 67 et 350 millions de dollars.
Le choléra affecte entre 1,3 et 4 millions de cas chaque année dans le monde, avec entre 21 000 et 143 000 décès. L'OMS a classé la résurgence mondiale du choléra comme une urgence de niveau 3, son niveau interne le plus élevé.
Jaguar Health (NASDAQ:JAGX) gab bekannt, dass die FDA dem Crofelem die Orphan-Drug-Designation (ODD) zur Behandlung von Durchfall bei Cholerapatienten erteilt hat. Dieses pflanzenbasierte rezeptpflichtige Medikament erhielt zuvor Phase-2-Daten aus klinischen Studien in Bangladesch. Die ODD bietet Entwicklungssanreize, darunter Steuervergünstigungen und einen siebenjährigen Marktexklusivitätszeitraum, falls genehmigt.
Crofelem ist derzeit an fünf klinischen Studien zu seltenen Krankheiten beteiligt, wobei die Patientendosierung für Dezember 2024 und das erste Quartal 2025 geplant ist. Ergebnisse könnten im zweiten Quartal 2025 verfügbar sein. Das Unternehmen plant auch, ODD für einen Arzneimittel der zweiten Generation, NP-300, zu beantragen, zusammen mit einem Priority Review Voucher für tropische Krankheiten, der historisch für 67 bis 350 Millionen US-Dollar verkauft wurde.
Cholera betrifft jährlich weltweit 1,3 bis 4 Millionen Fälle, mit 21.000 bis 143.000 Todesfällen. Die WHO hat den weltweiten Cholera-Rückfall als Notfall der Kategorie 3 klassifiziert, das höchste interne Niveau.
- FDA granted orphan-drug designation providing 7-year marketing exclusivity
- Five ongoing rare disease clinical trials with results expected Q2 2025
- Potential valuable Priority Review Voucher worth $67-350 million for NP-300
- Large global market opportunity with 1.3-4 million annual cholera cases
- Clinical trial results not yet available
- Additional regulatory approvals still needed
- Very low incidence of cholera in US market
Insights
The FDA's orphan-drug designation for crofelemer represents a significant milestone for Jaguar Health's treatment of cholera-related diarrhea. This designation provides important benefits including
The potential for a Tropical Disease Priority Review Voucher with NP-300 is particularly valuable, as these vouchers have historically sold for
For a small-cap company with a market cap of just
The global cholera treatment market, while in the US, represents a significant opportunity given WHO's grade 3 emergency classification and the disease's endemic status in multiple countries. The multiple clinical trials across various orphan indications also diversify risk and create multiple potential revenue streams.
World Health Organization (WHO) has classified the global resurgence of cholera at the highest internal level for emergencies; 1.3 to 4 million cholera cases and 21,000 to 143,000 cholera-related deaths occur each year worldwide
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 European Medicines Agency (EMA) for both short bowel syndrome and microvillus inclusion disease
SAN FRANCISCO, CA / ACCESSWIRE / December 17, 2024 / Jaguar Health(NASDAQ:JAGX) (Jaguar) family company Napo Pharmaceuticals (Napo) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation (ODD) to crofelemer, the company's novel plant-based prescription drug, for treatment of diarrhea in cholera.
"We are very pleased that crofelemer has been granted orphan-drug designation for this important indication," 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 qualifies the company for various development incentives, including tax credits for qualified clinical testing and relief of filing fees, and provides 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 for crofelemer for this indication from the EMA as well in the future."
Due to the very low incidence and prevalence of cholera in the United States, cholera is an orphan indication in this country. Crofelemer is also the subject of five other rare/orphan disease-related clinical efforts - three investigator-initiated trial (IIT) proof-of-concept studies and two Phase 2 studies - for the indications of short bowel syndrome with intestinal failure (SBS-IF) and/or microvillus inclusion disease (MVID) in the US, European Union, and/or Middle East/North Africa regions. Dosing of the first patient in each of these five studies is expected to occur throughout December 2024 and Q1 2025, with availability of IIT proof-of-concept results potentially in Q2 2025. In accordance with the guidelines of specific EU countries, published data from clinical investigations in SBS-IF and MVID could support reimbursed early patient access to crofelemer for these debilitating conditions in those countries.
Crofelemer has also been granted ODD by the FDA and the EMA for both MVID and SBS.
Additionally, Jaguar intends to pursue orphan-drug designation and a Tropical Disease Priority Review Voucher for the indication of treatment of diarrhea in cholera with a proprietary second-generation anti-secretory agent, NP-300, which, like crofelemer, is sustainably derived from the Croton lechleri tree. Priority review vouchers are transferable, and in past transactions by other companies have sold for values ranging from
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.1 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. From January 1, 2024 to July 28, 2024, a cumulative total of 307,433 cholera cases and 2,326 deaths were reported from 26 countries across five World Health Organization (WHO) regions.2 WHO classified the global resurgence of cholera as a grade 3 emergency in January 2023, the highest internal level for emergencies in WHO.2 Based on the number of outbreaks and their geographic expansion, alongside the shortage of vaccines and other resources, WHO continues to assess the risk at the global level as very high and the event remains classified as a grade 3 emergency.2
About Orphan-Drug Designation in the US and European Union
The Orphan Drug Act 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.
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. Napo 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 (Napo) focuses on developing and commercializing human prescription pharmaceuticals for essential supportive care and management of neglected gastrointestinal symptoms across multiple complicated disease states. Napo's crofelemer is FDA-approved under the brand name Mytesi® for the symptomatic relief of noninfectious diarrhea in adults with HIV/AIDS on antiretroviral 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
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Forward-Looking Statements
Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that the company will pursue ODD for crofelemer for treatment of diarrhea in cholera from the EMA in the future, Jaguar's expectation that dosing of the first patient in each of three IITs and two Phase 2 studies of crofelemer for SBS-IF and/or MVID will occur throughout December 2024 and Q1 2025, Jaguar's expectation that IIT proof-of-concept results for crofelemer for SBS-IF and/or MVID could potentially be available in Q2 2025, Jaguar's expectation that, in accordance with the guidelines of specific EU countries, published data from clinical investigations in SBS-IF and MVID could support reimbursed early patient access to crofelemer for these debilitating conditions in those countries, Jaguar's expectation that it will also pursue the indication of treatment of diarrhea in cholera with NP-300, Jaguar's expectation that it will pursue orphan-drug designation for NP-300 for treatment of diarrhea in cholera, and Jaguar's expectation that NP-300 may qualify for a Tropical Disease Priority Review Voucher if NP-300 is approved by the FDA for treatment of diarrhea in cholera. 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.
Contact:
hello@jaguar.health
Jaguar-JAGX
SOURCE: Jaguar Health, Inc.
View the original press release on accesswire.com
FAQ
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