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
Ant International Research Highlights How Readable AI Memories Can Unlock More Personalised, Privacy-conscious Consumer Experiences2.11.2026 09:52:00 CET | Press release
Human sales and customer representatives have institutional memories to serve clients: their preferences and choices. How can we build a memory mechanism for AI services to create similarly personalised experiences? Ant International’s research on this subject recently received the Senior Area Chair (SAC) Highlight Award from the Best Paper Committee at EMNLP 2026, a leading global AI conference. Why do we need “readable AI memory”? In this paper titled 'Text as a universal interface for transferable personalisation', we look into one fact: until now, AI memory of a user has usually been generated and stored as dense numerical code only the original system can interpret. Two long-standing problems with that: First, today's user profiles are "black boxes" — opaque code locked inside a single app, so activity in one service teaches nothing to another, and every product has to re-learn you from scratch. Second, profiles are welded to the model that created them, so they don't transfer whe
Liberis and Qred Complete Merger Following Regulatory Approval to Create Global SMB Financial Platform2.11.2026 09:00:00 CET | Press release
Key points The merger is complete following regulatory approval. The combined group serves more than 60,000 small businesses across 17 countries, with access to approximately 11.5 million addressable merchants. Emil Sunvisson becomes Group CEO and Rob Fairfield becomes Deputy CEO. Both brands, products, partner integrations and customer relationships continue as they do today. Liberis, a leading global embedded finance platform, and Qred, one of Europe’s foremost digital small business banks, today announced the completion of their merger, first announced in June 2026, following approval from the Swedish Financial Supervisory Authority (SFSA). The merger combines Qred’s regulated banking capability and direct relationships with small businesses with Liberis’s embedded finance technology and distribution through more than 30 partners, including Deliveroo, Vagaro and Nexi. The group serves more than 60,000 small businesses across 17 countries and has access to approximately 11.5 million
Car Connectivity Consortium Expands Focus on Digital Access for Two-Wheelers and Micromobility2.11.2026 09:00:00 CET | Press release
Ahead of EICMA 2026, CCC highlights industry collaboration around secure, interoperable digital access for motorcycles, scooters, and other two-wheeled vehicles The Car Connectivity Consortium® (CCC), the global organization advancing interoperable connectivity between vehicles and devices, today highlighted its expanding work across the two-wheeler and micromobility markets ahead of EICMA 2026 in Milan, Italy. Through its Light Motor & Mobility Task Group (LMTG), CCC is bringing member companies together to address the distinct digital access needs of these markets and identify opportunities to advance standardization and interoperability. Key highlights of CCC's two-wheeler and micromobility work include: Addressing two-wheeler digital access needs: LMTG is examining requirements across motorcycles, scooters, e-bikes and other two- and three-wheeled vehicles. Exploring new applications for digital key technology: Member discussions include vehicle access and starting, access to vehic
OpenGate Capital Completes the Acquisition of Maersk Training, the Global Safety Training and Gas Safety Services Business of Maersk2.11.2026 09:00:00 CET | Press release
OpenGate Capital (“OpenGate”), a global private equity firm, announced today that it has completed the acquisition of Maersk Training and its subsidiary Maersk H2S Safety Services. The company, a provider of global safety training and gas safety services, is being carved out from A.P. Møller - Mærsk A/S (MAERSK A:CPH), a listed integrated logistics company. Terms of the acquisition were not disclosed. Headquartered in Svendborg, Denmark, Maersk Training is a leading global provider of essential safety services and training. Through Maersk H2S Safety Services, the company provides specialized outsourced hydrogen sulphide and Health, Safety and Environment safety services for customers in high-risk operating environments. The H2S Safety Services team provides onsite supervision, monitoring and safety equipment maintenance where the safety of personnel and the protection of assets are critical. Through its training business, the company also delivers safety training, competency developmen
SES to Supply Finnair with Ground-like Connectivity Experience in the Skies2.11.2026 08:00:00 CET | Press release
Multi-orbit inflight connectivity service will deliver faster, more reliable onboard internet experience to 18 Embraer aircraft SES today announced a new agreement with Finnair, Finland's flagship airline, to equip 18 E195-E2s with multi-orbit in-flight connectivity, enabling Finnair’s customers to enjoy fast, reliable internet access throughout their journey. Under the agreement, the Embraer E2 aircraft, which will be line-fitted at Embraer, will be equipped with SES’s electronically steered array antenna (ESA) offering reliable connectivity to the SES multi-orbit network, combining SES’s geostationary (GEO) satellite services with low-earth orbit (LEO) capacity from its partner Eutelsat. The first connected aircraft is expected to enter service in 2027. “Inflight Wi-Fi is a core part of the travel experience,” said Blane Boynton, Senior Vice President Aviation at SES. “SES’s multi-orbit network delivers the reliability, performance and flexibility airlines need today while providing
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