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
IQM Demonstrates Entanglement Across a Complete 54-Qubit Processor, Bringing Full-Chip Performance to Cloud Users29.10.2026 08:00:00 CET | Press release
An automated state-preparation routine and enhanced calibration protocol keep fidelity above the entanglement threshold at full scale, and cloud users now have access to the same system performance. IQM Quantum Computers (Nasdaq: IQMX), a global leader in superconducting quantum computers, today announced that its cloud-accessible IQM Emerald system has fully entangled all 54 qubits on the chip simultaneously, a result that shows the system’s computational quality holds up when every qubit is put to work at once. IQM’s cloud customers now have access to that same full-chip performance, so their computations can draw on the entire processor rather than a fraction of it. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261029191153/en/ IQM measured a fidelity of 0.515 at 40 qubits without readout error mitigation (REM) and 0.516 at the full 54 qubits with REM applied, both above the classical threshold of 0.500 that separates ge
Takeda Announces FY2026 First-Half Results Highlighting Launch Momentum and Pipeline Milestones29.10.2026 07:36:00 CET | Press release
ORZEYFULTM approved in the U.S., Japan and China for narcolepsy type 1 (NT1), MIMRYLOTM launched in the U.S. for erythrocytosis in polycythemia vera (PV)Zasocitinib advances to U.S. Food & Drug Administration (FDA) Priority Review and European Medicines Agency (EMA) reviewLate-stage pipeline continues to advanceFY2026 full-year outlook upgraded to reflect foreign exchange (FX) benefitTakeda will host a Capital Markets Day on Dec. 11, 2026, in Tokyo, Japan Takeda (TOKYO:4502/NYSE:TAK) announced financial results for the first half of fiscal year 2026 (April 1, 2026, to Sept. 30, 2026). The period included the approval of ORZEYFUL (oveporexton) in the U.S., Japan and China, the U.S. launch of MIMRYLO (rusfertide), progress in the regulatory review of zasocitinib, and additional clinical evidence across the late-stage pipeline. Takeda’s focus remains on execution against the first horizon of its growth strategy: successfully launching three new medicines, advancing its late-stage pipeline
AB InBev Reports Third Quarter 2026 Results29.10.2026 07:01:00 CET | Press release
Solid top- and bottom-line performance with volume growth, revenue up 4.9% and Underlying EPS up 22% Anheuser-Busch InBev (Brussel:ABI) (BMV:ANB) (JSE:ANH) (NYSE:BUD): This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261028546637/en/ Regulated and inside information1 “Delivering solid growth today while investing for tomorrow - our portfolio of megabrands and mega platforms continues to win with consumers, and our performance this quarter reflects the strength of our diversified footprint, best-in-class capabilities and consistent execution of our strategy. Thank you to our colleagues around the world for their commitment and dedication as we continue to compound growth and create long-term value.” – Michel Doukeris, CEO, AB InBev Revenue +4.9% Revenue increased by 4.9% in 3Q26 with revenue per hl growth of 4.2% and by 5.4% in 9M26 with revenue per hl growth of 4.3%. Reported revenue increased by 9.1% in 3Q26 to 16 512 million
EIG and Acwa Announce Plenum Power, a Strategic Partnership to Invest in U.S. Power Infrastructure29.10.2026 06:00:00 CET | Press release
Partnership combines EIG’s platform-building, investment and financing expertise with Acwa’s large-scale power development and operating capabilities and is targeting a launch with approximately $4 billion of initial equity commitment EIG, a leading institutional investor in the global energy and infrastructure sectors, and Saudi-listed Acwa, one of the world's largest independent power producers, today announced that they have entered into definitive agreements to establish Plenum Power, a strategic partnership focused on investment opportunities in the United States. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261028377569/en/ Dr. Samir J. Serhan, Chief Executive Officer of Acwa (left), and R. Blair Thomas, Chief Executive Officer of EIG (right) Building on an established relationship that includes prior collaboration on projects in the Middle East, EIG and Acwa are now partnering to pursue opportunities in the U.S. pow
Murata Launches World’s Smallest RF / Microwave Coaxial Connectors with a Switch, Cutting PCB Mounting Area by Approximately 40%29.10.2026 03:00:00 CET | Press release
Murata Manufacturing Co., Ltd. (TOKYO: 6981) (ISIN: JP3914400001) has begun mass production of the SWL series MM8330-2600RJ5 and MM8330-2600RK20, the world’s smallest RF / microwave coaxial connectors with a switch*. Measuring just 1.0 mm × 1.0 mm × 0.55 mm, the new connectors reduce PCB mounting area by approximately 40% compared with Murata’s previous SWJ series. When used with dedicated probes, the SWL series reduces its PCB footprint, including the keep-out area, by approximately 60% compared with Murata’s previous SWJ series. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20261028261665/en/ [Murata Manufacturing Co., Ltd.] RF / microwave coaxial connectors with a switch As wireless devices incorporate more functionality, rising component counts are placing increasing pressure on limited PCB space. RF test connectors require not only mounting area for the connector itself, but also sufficient clearance around the component
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