Vivet’s Second Gene Therapy Product, VTX-803 for PFIC3, Receives US and European Orphan Drug Designation
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.
Media & Investor:
Business Development Director
About Business Wire
(c) 2018 Business Wire, Inc., All rights reserved.
Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.
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
rf IDEAS ® Adds HID ® Mobile Access to Its WAVE ID ® Mobile Offering6.7.2020 22:57:00 CEST | Press release
rf IDEAS, a leading manufacturer of credential readers for logical access and authentication, continues to advance the future of secure mobile access. Developed in partnership with HID Global, the newest addition to the rf IDEAS WAVE ID Mobile reader line supports the HID Mobile Access credential on Bluetooth® Low Energy enabled mobile devices. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200706005578/en/ WAVE ID® Mobile for HID® Mobile Access (Graphic: Business Wire) The WAVE ID Mobile for HID Mobile Access provides smartphone users with contactless logical access to workstations, networks, applications, secure printers and more via the HID credential. This new reader also provides support for nearly any physical proximity or contactless smart card in use worldwide. The HID Mobile Access credential can be obtained from HID Global through rf IDEAS. It is based on the highly secure SEOS® platform and delivered through an ea
New UC Berkeley Global Program for Freshmen Offers an Online “Study-Abroad” Option6.7.2020 19:40:00 CEST | Press release
UC Berkeley Global is proud to announce its Global Program for Freshmen (GPF), launching Fall 2020. This unique “online study-abroad” program is designed for incoming first-year university students seeking a close-knit, collaborative learning community within a digital-learning environment. Students will have access to Berkeley instructors and mentors while working closely with their peers in small cohorts using online tools. GPF offers courses in Letters & Science (L&S) that cover key prerequisites for majors across various disciplines. Elective courses allow students to complete a total of 15 credits per semester, while additional programming will focus on important issues for first-year students, including preparing for college life, selecting a major and career development. Upon completion, students earn credits that they can transfer to their home institution. This makes GPF an ideal opportunity for universities seeking a high-quality online option for their incoming students, or
Research Enhances Understanding of the Nutritional and Economic Differences in Soybean Meal from Different Origins6.7.2020 16:17:00 CEST | Press release
A new meta-analytical study reinforces U.S. Soy’s reputation for being a global leader in quality and nutrient-density. The study, entitled, “Chemical composition, protein quality and nutritive value of commercial soybean meals produced from beans from different countries,” demonstrates that not all soybean meals are created equal and that meal from different countries of origin should be treated individually when formulating swine and poultry diets. The data from this study were then processed by the Nutrient Value Calculator (NVC), a software tool constructed by Genesis Feed Technologies, a company built to bring visibility into feed costs. This cost analysis supported the economic benefits of U.S. soybean meal relative to meal from other origins as a key ingredient in poultry rations. Dr. Gonzalo Mateos, Professor of Animal Science at the University of Madrid in Spain and study co-author, first presented findings from the meta-analysis at the U.S. Soybean Export Council’s (USSEC) As
Echosens Announces NICE, UK Health Technology Assessment Organization, Issues MedTech Innovation Briefing on FibroScan Liver Exam in Primary Care6.7.2020 15:00:00 CEST | Press release
Echosens, a high-technology company offering the FibroScan family of products, announces today that the National Institute for Health and Care Excellence (NICE), the United Kingdom’s health technology assessment body providing national guidance and advice to improve health and social care, issued a MedTech Innovation Briefing (MIB) dedicated to utilization of FibroScan non-invasive technology in the primary care setting. The NICE experts concurred that this approach could provide a cost-effective way for the National Health System (NHS) to assess liver fibrosis and cirrhosis in primary care--reducing the need for treatment, referrals and biopsies, and improving accuracy and speed. “Using FibroScan as an interventional therapy helps primary care physicians and nurse specialists engage patients by demonstrating the results and outcomes as a value change that patients can visualize on a regular basis and give them confidence that their efforts have an impact,” says Louise Campbell, patien
WEP Clinical Partners With Nabriva Therapeutics for the Named Patient Supply of XENLETA ® (lefamulin)6.7.2020 13:00:00 CEST | Press release
WEP Clinical (WEP), a specialist pharmaceutical services company, has signed an exclusive agreement with Nabriva Therapeutics (Nabriva) to supply XENLETA® (lefamulin) on a named patient or expanded access basis. The Named Patient Program (NPP) is designed to ensure that physicians, contingent on meeting the necessary eligibility criteria and receiving approval, can request IV or oral XENLETA on behalf of patients who live in certain countries where it is not yet available. XENLETA was discovered and developed by Nabriva (NASDAQ: NBRV), a commercial-stage biopharmaceutical company focused on commercialization and development of anti-infective agents. It is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with community-acquired bacterial pneumonia (CABP) caused by susceptible microorganisms. XENLETA is the first and only oral and IV pleuromutilin empiric antibiotic that acts against the pathogens that commonly cause CABP. Nabriva announced on May 29th
Pudu Robotics Raised over $15M Series B Financing with Meituan as the Exclusive Investor6.7.2020 11:11:00 CEST | Press release
On July 1, 2020, Pudu Robotics, an indoor delivery robot provider, officially announced the completion of B round financing of more than $15M with Meituan as the exclusive investor. Zhang Tao, CEO of Pudu Robotics, said the fund would be mainly used to develop products, expand sales, explore new usage scenarios and expand overseas market. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200706005180/en/ (Photo: Business Wire) Pudu Robotics is a top-notch intelligent indoor delivery robot provider in China. The company focuses on the R&D, design and production of robots and offers a number of popular delivery robots, including PuduBot (food delivery robot), BellaBot (food delivery robot), HoloBot (building delivery robot), HolaBot (dish-return robot), and GazeBot (multi-scene delivery robot). Its first mass-produced robot PuduBot has been widely used in extensive scenarios, including restaurants, hotels, office buildings, hospi