VIVET-THERAPEUTICS
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
Most Delivered Light-Entry Jet Cessna Citation M2 Gen2 With Garmin Autothrottles Enters Into Service, Bringing Greater Control and Precision to Pilots14.1.2026 20:30:00 CET | Press release
The Cessna Citation M2 Gen2 with Garmin Autothrottles recently entered into service. Certified by the Federal Aviation Administration in October 2025, the integration of Garmin’s Autothrottle system in the Citation M2 Gen2 further supports the most delivered light-entry jet’s capabilities by bringing greater control and precision to pilots. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260114646844/en/ Most delivered light-entry jet Cessna Citation M2 Gen2 with Garmin Autothrottles enters into service, bringing greater control and precision to pilots (Photo Credit: Textron Aviation). The Cessna Citation M2 Gen2 is designed and manufactured by Textron Aviation Inc., a Textron Inc. (NYSE: TXT) company. “We continue to invest in our products to provide customers with the best aviation experience in the world," said Lannie O’Bannion, senior vice president, Sales & Marketing. “The M2 Gen2 leads its segment as the most delivered
TwentyTwo Real Estate and Farallon Capital Complete €761 Million Investment in Isemia, a €1.2bn Healthcare Real Estate Company Created With emeis14.1.2026 18:30:00 CET | Press release
TwentyTwo Real Estate, an independent European real estate investment and management firm, announces the completion, alongside Farallon Capital, of a €761 million investment that led to the creation of Isemia (www.isemiabyemeis.com), a leading healthcare real estate company holding a €1.2 billion portfolio of assets operated by emeis. The transaction represents a significant milestone in TwentyTwo Real Estate’s long-term investment strategy focused on healthcare and operational living real estate. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260114262830/en/ Fachklinik Schömberg Isemia comprises 68 high-quality assets across France, Germany and Spain, with a balanced mix of nursing homes (48%) and clinics (52%). The portfolio benefits from strong fundamentals and resilient operating performance. All properties will continue to be operated by emeis under long-term lease agreements, ensuring continuity of care for residents
AI’s Water Demand to Surge Nearly 130% by 2050 – New Research Shows How to Build a Water-Secure AI Economy14.1.2026 16:00:00 CET | Press release
Analysis finds AI could add 30 trillion liters of annual water demand, but targeted reuse and digital infrastructure investments can substantially offset demand and protect communities The rapid global expansion of artificial intelligence is set to drive a 129% increase in water demand across the AI value chain by 2050, according to new research by Xylem (NYSE: XYL) and Global Water Intelligence (GWI). Watering the New Economy: Managing the Impacts of the AI Revolution provides the most comprehensive assessment to date of how AI is reshaping global water use. It also highlights practical ways to meet that demand while strengthening water security for communities. Spending on AI is forecast to approach $2 trillion in 2026,1 accelerating infrastructure build-out worldwide. By the middle of the century, this expansion is projected to add 30 trillion liters of water demand, annually, fueled by a surge in power generation (~54% of the increase), semiconductor fabrication (~42%), and data ce
ACCELQ Crowned Leader and Customer Favorite in The Forrester Wave™ for Autonomous Testing Platforms14.1.2026 16:00:00 CET | Press release
Agentic Test Automation Pioneer Caps Record-Breaking Year of Growth and "AI-Based Engineering Solution of the Year" Honors Forrester has named ACCELQ a Leader in The Forrester Wave™: Autonomous Testing Platforms, Q4 2025. This marks the third consecutive Wave in which ACCELQ has been recognized as a Leader. Notably, this evaluation placed strong emphasis on generative AI innovation and real-world customer adoption. ACCELQ received the highest scores for both capabilities and vision and was the only platform to be recognized as a Customer Favorite, earning a "double halo" for exceptional customer endorsement. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260113112861/en/ ACCELQ recognized as a Leader and Customer Favorite in The Forrester Wave™️: Autonomous Testing Platforms, Q4 2025, validating its agentic, GenAI-embedded testing vision. According to the Forrester report, ACCELQ received the highest score among all evaluate
Primient to Acquire Full Ownership of Primient Covation, LLC14.1.2026 16:00:00 CET | Press release
Primient, a global leader in biomanufacturing, announced today it has agreed to purchase the remaining ownership interest in Primient Covation, LLC (“Primient Covation”), establishing Primient as the sole owner of the company and marking an exciting new chapter in Primient’s growth in the bioeconomy. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260114344080/en/ This acquisition is the next step of Primient’s strategy to strengthen Primient’s leadership position in biomanufacturing and builds upon recent announcements, including Primient’s partnership with Sustainea and the creation of the iPROOF venture. Primient Covation stands as a leading force in the U.S. biomanufacturing sector, distinguished by its broad range of biomanufacturing and fermentation expertise. The company was founded in 2000 to supply a variety of end markets, including cosmetics and personal care, textiles, fashion, coatings, functional fluids, and oth
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