Jaguar Health планирует подать заявку на регистрацию крофелемера для лечения редкого детского заболевания в 2027 году

Jaguar remains sharply focused on its ongoing global development program for oral crofelemer as adjunctive therapy to parenteral support (PS) for pediatric and adult rare IF diseases, including microvillus inclusion disease (MVID) Company plans to file NDA for crofelemer for MVID in mid-2027 to obtain FDA approval SAN FRANCISCO, CA / ACCESS Newswire / September 4, 2026 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) today announced that Jaguar family company Napo Pharmaceuticals (Napo) is providing the company's novel crofelemer powder for oral solution for treatment of intestinal failure (IF) in an infant patient due to congenital sodium diarrhea (CSD), which is caused by a mutation in the GUCY2C gene. Napo remains sharply focused on its ongoing global development program for oral crofelemer as adjunctive therapy to parenteral support (PS) for pediatric rare IF diseases, including microvillus inclusion disease (MVID) and short bowel syndrome with IF (SBS-IF). In June 2026, as announced, data was presented at the 58th Annual European Society for Pediatric Gastroenterology, Hepatology & Nutrition (ESPGHAN) Meeting in Lille, France from ongoing investigational studies with oral liquid crofelemer in pediatric patients with IF due to MVID and SBS-IF. The results of ongoing investigations of liquid oral crofelemer formulations demonstrate substantial reductions in weekly PS needs as well as in PS normalized to body weight in pediatric IF patients dosed orally for more than one year, with no significant clinical or laboratory abnormalities. In one MVID patient, the weekly PS requirements normalized to body weight have been reduced by up to 48% following >12 months of liquid oral high-dose crofelemer therapy. In two pediatric SBS-IF patients, weekly PS requirements normalized to body weight were reduced up to 40% following >12 months of liquid oral high dose crofelemer therapy. IF patients, including those with MVID or short bowel syndrome (SBS), cannot orally absorb adequate nutrients, electrolytes and fluids, and require lifelong total parenteral nutrition (TPN) with or without supplemental intravenous fluids, which together comprise parenteral support (PS), for up to seven days per week and up to 20+ hours per day. While PS is supportive, palliative and lifesaving, it is associated with serious, often fatal complications. Patients with GUCY2C mutation and CSD have a significant challenge for managing nutritional and electrolyte deficiencies - especially pediatric patients, given the higher impact on growth and developmental status. This pediatric patient with IF due to CSD requires significant daily PS and is receiving crofelemer as adjunctive therapy to the PS to reduce their daily and weekly PS needs, as observed in pediatric IF patients with MVID and SBS. "Napo remains committed to supporting physicians who determine that the unmet medical need for their specific pediatric IF patients could be mitigated through the use of our novel crofelemer formulation as an adjunctive oral therapy to PS," said Pravin Chaturvedi, PhD, Napo's and Jaguar's Chief Scientific Officer and Chair of the Scientific Advisory Board. "We wish these physicians and their patients the best outcomes and are grateful for their collaboration. Liquid oral crofelemer as adjunctive therapy to PS has been well tolerated with no significant clinical or laboratory abnormalities throughout the ongoing treatment period of >12 months in selected pediatric IF patients with MVID and SBS-IF. In addition, the reductions in weekly PS requirements normalized to body weight with excellent tolerability support the continued development of liquid oral crofelemer as the first oral adjunctive therapy for pediatric IF patients receiving long-term and/or lifelong PS." "We're very pleased to be able to make our novel liquid oral formulation of crofelemer available to this infant for compassionate use," said Lisa Conte, Jaguar's founder, president, and CEO. "We remain sharply focused and committed to our global IF development program for our novel liquid oral crofelemer formulation as adjunctive therapy for the management of rare IF disorders. Our IF program is expected to continue to provide clinical milestones and is the subject of business development discussions with the potential to bring in non-dilutive funds from potential licensee partners. If appropriate, Jaguar plans to apply for Breakthrough Therapy Designation for crofelemer for the indication of MVID, with a planned filing of an NDA (New Drug Application) with the U.S. Food and Drug Administration (FDA) for this indication in mid-2027. We have completed the randomized double-blind treatment phase in our pivotal clinical trial of the novel liquid oral formulation of crofelemer in pediatric MVID patients. Upon completion of the trial, data from this study may serve as the basis for a Breakthrough Therapy Designation application. Because MVID is a devastating ultrarare pediatric disorder with a lethal natural history - and an estimated worldwide prevalence of only 200 patients - a trial of crofelemer in just a small number of MVID patients is expected to be clinically meaningful and significant." About Crofelemer Crofelemer is a novel, oral plant-based prescription medicine purified from the red bark sap, also referred to as "dragon's blood," of the Croton lechleri tree in the Amazon Rainforest. 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. Jaguar family companies Napo Pharmaceuticals, Inc. (Napo) and Napo Therapeutics S.p.A. focus on the development and commercialization of novel crofelemer powder for oral solution for the treatment of rare and orphan gastrointestinal disorders with intestinal failure, including microvillus inclusion disease and short bowel syndrome. For more information about: Jaguar Health, visit https://jaguar.health Napo Pharmaceuticals, visit napopharma.com Napo Therapeutics, visit napotherapeutics.com Forward-Looking Statements Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that its intestinal failure program will continue to provide clinical milestones, Jaguar's expectation that the opportunity may exist to bring in non-dilutive funds from potential licensee partners to support the intestinal failure program, Jaguar's plans to pursue Breakthrough Therapy designation for crofelemer for the indication of MVID, Jaguar's expectation that the company will file an NDA with the FDA for the MVID indication in mid-2027, and Jaguar's expectation that a trial of crofelemer in a small number of MVID patients may be statistically meaningful. 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 several 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: [email protected] Jaguar-JAGX SOURCE: Jaguar Health, Inc. View the original press release on ACCESS Newswire: https://www.accessnewswire.com/newsroom/en/healthcare-and-pharmaceutical/additional-infant-patient-to-receive-jaguar-healths-crofelemer-for-co-1216539