VIVET-THERAPEUTICS
1.6.2020 13:02:07 CEST | Business Wire | Press release
Vivet Therapeutics announced today that both the Food and Drug Administration (FDA) and the European Commission (EC) have granted Orphan Drug Designation (ODD) for Vivet’s second gene therapy product, VTX-803, for the treatment of Progressive Familial Intrahepatic Cholestasis type 3 (PFIC3). PFIC3 is a rare life-threatening and chronically debilitating condition due to progressive severe liver dysfunction, accompanied by jaundice, portal hypertension, hepatosplenomegaly and failure to thrive. The symptoms of the disease usually appear first in childhood with progressive cholestasis, evolving to hepatic failure, cirrhosis and need for liver transplantation.
“We are very pleased to receive these designations which further validate Vivet’s efforts to treat rare inherited metabolic disorders. The FDA and EC have both recognized the unmet need for a safe and effective treatment of PFIC3 and the potential of VTX-803 to address such need. The field of Progressive Familial Intrahepatic Cholestasis has been gaining increased attention in the medical field with numerous ongoing clinical trials. There is a high unmet medical need, especially in the paediatric segment, for this condition, for which liver transplantation remains the only truly curative option today” stated Jean-Philippe Combal, co-founder & CEO of Vivet Therapeutics.
VTX-803 is Vivet’s second gene therapy product to be granted Orphan Drug Designation. This is an important value driver for Vivet which would provide 10 & 7 years of market exclusivity in the EU & US respectively if VTX-803 is approved for the treatment of PFIC3. VTX-803 is currently under IND-enabling studies and initiation of clinical development is planned for the second part of 2021 in both the US and the EU.
“We are very happy about this recognition of our scientific achievement for a complex liver disease and further validation of our strategic collaboration with Fundación para la Investigación Médica Aplicada (FIMA). This program could be a game-changer in the gene therapy field for PFIC disorders” added Dr. Gloria González-Aseguinolaza, Vivet Therapeutics co-founder & CSO and deputy director at CIMA.
VTX-803 was recently the subject of an oral presentation at 2019 ASGCT (American Society of Gene and Cell Therapy), a lecture at the 2019 ESGCT (European Society of Gene and Cell Therapy) annual meetings and a publication in Nature Communications (Weber et al. Nature Communications, 2019, 10(1):5694).
About Vivet Therapeutics
Vivet Therapeutics is an emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.
Vivet is building a diversified gene therapy pipeline based on novel adeno-associated virus (AAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigación Medica Aplicada, University of Navarra based in Pamplona, Spain.
Vivet’s lead program, VTX-801, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation by the Food and Drug Administration and the European Commission.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200601005221/en/
About Business Wire
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Esentia Announces Successful Pricing of 6.125% Senior Notes Due 2033 and 6.500% Senior Notes Due 20388.5.2026 03:24:00 CEST | Press release
Esentia Energy Development, S.A.B. de C.V. (“ESENTIA” or the “Company”), today announced the pricing of U.S.$1,000,000,000.00 aggregate principal amount of its 6.125% Senior Notes due 2033 (the “2033 Notes”) and U.S$1,000,000,000.00 aggregate principal amount of its 6.500% Senior Notes due 2038 (the “2038 Notes” and, together with the 2033 Notes, the “Notes”) to be issued by the Company in a private offering to qualified institutional buyers in accordance with Rule 144A under the Securities Act of 1933, as amended (the “Securities Act”), and outside the United States to non-U.S. persons in accordance with Regulation S under the Securities Act. The 2033 Notes will be issued at a price of 99.517%, and the 2038 Notes will be issued at a price of 98.444%. The 2033 Notes mature on July 30, 2033, and the 2038 Notes mature on July 30, 2038, and will be fully and unconditionally guaranteed by certain of the Company's subsidiaries. The settlement of the Notes is expected to take place on May 14
83% of Restaurants Are Invisible in AI Search: New Uberall Report Reveals the Discovery Gap Reshaping the Quick Service Restaurant Industry7.5.2026 19:35:00 CEST | Press release
Industry-first benchmark study analyzes how ChatGPT, Gemini, Perplexity, Copilot and Google AI Overviews recommend restaurants Uberall, the global leader in location marketing technology, today released Fast Food, Faster Discovery: The 2026 GEO Playbook for Multi-Location QSRs — the industry’s first benchmark report measuring how AI assistants recommend restaurants and how multi-location QSR (Quick-Service Restaurant) brands can adapt their local marketing strategies for AI-mediated search. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260507962493/en/ Fast Food, Faster Discovery: Why AI Is the New Drive-Thru The report draws on Uberall’s proprietary GEO Studio benchmark data and aggregated performance metrics from its global QSR customer base. Its central finding: as consumer restaurant discovery rapidly shifts from traditional search to AI assistants, the majority of QSR locations are effectively absent from AI-generated
Department of Health - Abu Dhabi and ŌURA Partner to Advance Preventive Health in Abu Dhabi7.5.2026 18:57:00 CEST | Press release
Initially focused on women’s health, the partnership will unite Department of Health - Abu Dhabi’s longitudinal health data with ŌURA’s real-time insights, underpinned by a rigorous joint data governance framework Department of Health—Abu Dhabi (DoH) and ŌURA, maker of the world’s leading smart ring, Oura Ring, today announced a joint research programme to study how continuous wearable data can advance preventive health. The collaboration marks the beginning of a long-term partnership to advance Abu Dhabi’s prevention-first, data-driven healthcare agenda, and will initially focus on women’s health and cardiometabolic risk. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260507073495/en/ HE Mansoor Al Mansoori (Left) and Tom Hale (Right) The collaboration builds on Abu Dhabi’s advanced public health infrastructure and longitudinal health data assets, integrating them with ŌURA’s real-time, continuous insights across key health
Rave Sues Apple in Five Countries Over App Store Removal7.5.2026 17:03:00 CEST | Press release
Alleges Antitrust Violations in U.S., Canada, Brazil, the Netherlands and Russia to Restore Access for Users and Defend Fair Competition for App DevelopersAlleges Apple Acted to Stop Rave from Competing with Apple’s Own Co-Viewing AppRave Launches Proprietary, AI-enabled Moderation System, a-eye.com, to Protect Users from Explicit Content Rave Inc. (“Rave” or the “Company”), the developer and operator of the Rave app, a cross-platform co-viewing “super app” with more than 225 million downloads, today announced that it has filed antitrust lawsuits against Apple in five countries: the United States, Canada, Brazil, the Netherlands and Russia. The lawsuits challenge Apple’s unilateral and anticompetitive decision to remove Rave from its App Store, thereby distorting competition, reducing consumer choice in co-viewing functionalities and increasing the costs to users of switching between iPhone and competing smartphone devices. Rave operates a social entertainment app that allows users in
emnify named a Visionary in the 2026 Gartner® Magic Quadrant™ for Managed IoT Connectivity Services, Worldwide7.5.2026 17:00:00 CEST | Press release
First-time positioned vendor emnify recognized for its Completeness of Vision and Ability to Execute emnify, a global provider of cloud-native IoT connectivity solutions, today announced it has been recognized as a Visionary in the 2026 Gartner® Magic Quadrant™ for Managed IoT Connectivity Services, Worldwide. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260507642325/en/ emnify named a Visionary in the 2026 Gartner® Magic Quadrant™ for Managed IoT Connectivity Services, Worldwide. Get the report As a first-time recognized vendor, emnify believes this recognition reflects its forward-looking approach to IoT connectivity, building a platform designed to anticipate where enterprise needs are heading rather than replicating the connectivity models of the past. "We feel being named a Visionary in our first appearance in the Gartner® Magic Quadrant™ recognizes our deliberate strategy of the last decade: from owning our core netw
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.
Visit our pressroom