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
REPLY: A Face Only A Mother Could Love by Robert Gaudette Wins the Reply AI Film Festival 2026, the International Competition Exploring the Intersection of Cinema and Artificial Intelligence7.9.2026 12:02:00 CEST | Press release
Gabriele Salvatores chaired the international jury that selected the winner from more than 3,000 short films submitted from 76 countries. The awards ceremony took place in Venice on Sunday 6 September, alongside the 83rd Venice International Film Festival of La Biennale. Canadian filmmaker Robert Gaudette, with his short film A Face Only A Mother Could Love, has won the Reply AI Film Festival 2026, the international competition created by Reply to explore new frontiers in cinematic language through the use of artificial intelligence. The award was presented on Sunday 6 September in Venice, during a ceremony organised by Reply and Mastercard at the Match Point Arena, set up at Tennis Club Venezia on the Lido, alongside the 83rd Venice International Film Festival. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260907478421/en/ Canadian filmmaker Robert Gaudette, with his short film A Face Only A Mother Could Love, has won the
From China’s Mountains to the World’s Mountains: KAILAS Deepens Its Global Expansion Strategy7.9.2026 11:58:00 CEST | Press release
In late August 2026, KAILAS FUGA, the trail-running brand of KAILAS, a China-based professional outdoor brand, officially opened its first European store in Chamonix, France. As KAILAS’s first directly operated international store, the new location not only gives the brand a more direct presence in the European market, but also marks a new stage in its global expansion — moving from years of investment in products, events, athletes and international channels toward more localized operations and deeper brand development. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260906651659/en/ KAILAS First European Store in Chamonix Founded in 2003, KAILAS is one of the world’s three leading full-category climbing brands. Over more than two decades, the company has built its professional capabilities through product development, professional athletes, events and testing in real mountain environments. From mountaineering, rock climbing
Falcon Is Sold for AED 2.1 Million (USD 571,000) in Abu Dhabi, Setting the World’s Highest Price Ever at Falcon Auction7.9.2026 02:07:00 CEST | Press release
A Gyr Pure Ultra White falcon achieved a record-breaking sale of AED 2.1 million (USD 571,000) at the final falcon auction of the Abu Dhabi International Hunting and Equestrian Exhibition (ADIHEX) 2026, making it the highest value a falcon has ever been auctioned for. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260906974005/en/ The Gyr Pure Ultra White falcon sold for AED 2.1 million (USD 571,000) at the final falcon auction of ADIHEX 2026 in Abu Dhabi. (Photo: AETOSWire) Held under the patronage of His Highness Sheikh Hamdan bin Zayed Al Nahyan, Ruler’s Representative in Al Dhafra Region and Chairman of the Emirates Falconers’ Club, ADIHEX 2026 is organised by ADNEC Group in strategic partnership with the Emirates Falconers’ Club. Bidders competed for the prized Gyr Pure Ultra White falcon, which originated from the United States, with the winning bid setting the highest price a falcon has ever been auctioned for. The Gy
Ant International Secures Payment Institution Licence from the Central Bank of Brazil4.9.2026 16:41:00 CEST | Press release
Expanding the company’s capability to support local partners with FinAI solutions Ant International, the leading global digital payment, digitisation, and FinAI solutions provider, today announced that it has received a Payment Institution (PI) licence from the Central Bank of Brazil. “With the Central Bank's invaluable support, we are ready to step up more reliable and comprehensive global payment and account services to enterprises, platforms and SMEs in the country's dynamic digital economy, in ever deeper collaboration with its highly developed payments ecosystem and financial services industry,” said Rodrigo Reif, General Manager, Ant International Brazil. Ant International provides cross-border payments and account services, embedded credit and treasury services, and other finAI and blockchain solutions to clients across Asia, Europe and the Americas. The company processes over 20 million transactions daily, connecting 2 billion consumer accounts to 150 million global merchants v
Bending Spoons completes the acquisition of Airtable4.9.2026 14:30:00 CEST | Press release
Bending Spoons S.p.A. (NASDAQ: BSP) has completed the previously announced acquisition of Airtable, the platform used by more than 500,000 organizations to reshape how teams organize data and manage critical workflows. “Airtable continues to grow because customers value its best-in-class flexibility,” said Luca Ferrari, Bending Spoons CEO and co-founder. “Teams can bring all their data, context, and AI agents together in one place, shape their workflows around their specific needs, and progressively expand their use of the platform over time. As we take the reins, our focus will be on sustaining that growth by investing heavily in Airtable’s product, customer support, and go-to-market capabilities.” “I’m proud of what the Airtable team has built and excited to see the business enter this next chapter,” said Howie Liu, co-founder of Airtable. “I’m confident Bending Spoons has the resources and long-term perspective to help Airtable continue delivering exceptional value to its customers
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