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
INNIO Group Announces Filing of Registration Statement for Proposed Initial Public Offering11.5.2026 21:49:00 CEST | Press release
INNIO Group ("INNIO"), a leading global distributed energy solutions provider, today announces that it has publicly filed a registration statement on Form S-1 with the U.S. Securities and Exchange Commission ("SEC") relating to a proposed initial public offering of its common shares. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260511389675/en/ INNIO Group Announces Filing of Registration Statement for Proposed Initial Public Offering The timing of the offering, number of shares to be offered and the price range for the proposed offering have not yet been determined. INNIO has applied to list its common shares on the Nasdaq Global Select Market under the ticker symbol "INIO." Goldman Sachs & Co. LLC, J.P. Morgan and Morgan Stanley are acting as joint lead book-running managers for the proposed offering. BofA Securities, Barclays and Citigroup are acting as book-running managers for the proposed offering. Baird, BNP Paribas
Only 7% of Companies Achieve Full Compliance as Global Expansion Increases Legal Complexity11.5.2026 15:00:00 CEST | Press release
47% of general counsels say beneficial ownership rules pose the biggest risks to legal operations44% lack confidence in meeting cross-border data security requirements As businesses accelerate their global expansion in 2026, compliance fails to keep pace. In fact, only 7% of organizations report full compliance across their global entities, according to a new study by CSC, the leading provider of global business administration and compliance solutions. CSC surveyed 350 general counsel (GCs) and senior legal professionals across Europe, North America, and Asia Pacific to examine how their teams navigate international expansion, regulatory pressure, and the increasing adoption of artificial intelligence (AI).¹ The findings appear in CSC’s latest report, General Counsel Barometer 2026: From Complexity to Control. Most organizations report partial compliance, with over half (53%) estimating they are 50–75% compliant, and a further 35% placing themselves between 76%–99%. This leaves just 7%
IFF Opens Vanilla Innovation Center in Madagascar11.5.2026 14:15:00 CEST | Press release
Advancing science‑led flavor innovation where vanilla is grown IFF (NYSE: IFF)—a global leader in flavors, fragrances, food ingredients, health & bioscience—today announced the opening of its Vanilla Innovation Center in Madagascar, reinforcing vanilla as a strategic and priority tonality for IFF and strengthening its ability to innovate at origin. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260508110162/en/ IFF's Vanilla Innovation Center in Madagascar “The opening of the center marks an important step in how we approach vanilla innovation,” said Adam Jańczuk, Ph.D., senior vice president, research, creation and design, Taste, IFF. “By strengthening our presence at origin, we connect science, creativity and sustainability more closely, responding to climate changes, safeguarding quality and creating value across the supply chain.” Located in Toamasina, Madagascar’s principal seaport, near vanilla growing areas and post‑h
ARIS Recognised as a Leader in Gartner® Magic Quadrant™ for Process Intelligence Platforms, Believes This Reinforces Its Role in Enabling Enterprise AI at Scale11.5.2026 14:00:00 CEST | Press release
ARIS, the process context foundation platform for enterprise AI deployment, today announced its recognition as a Leader in the Gartner® Magic Quadrant™ for Process Intelligence Platforms. This is the fourth consecutive year that ARIS has been recognized as a Leader in the report and the company believes it underscores a continued commitment to innovation and growth as enterprises focus on turning AI ambition into measurable business outcomes. While technology has advanced rapidly, companies are struggling to operationalise AI across complex operating models. ARIS sees this recognition by Gartner as a reflection of its strength in delivering a single unified platform for process intelligence, providing the context layer on which G2000 organisations can successfully deploy and scale agentic AI. “AI is moving from experimentation to execution – but many enterprises are finding it difficult to scale,” said Guillaume Bacuvier, CEO of ARIS. “The reason is simple: AI lacks the context it need
HistoSonics Moves to Advance Additional Histotripsy Applications Announcing FDA Submission for Kidney Tumors11.5.2026 14:00:00 CEST | Press release
HistoSonics, the developer of the Edison® Histotripsy System and novel histotripsy therapy platform, today announced it has submitted a De Novo request to the U.S. Food and Drug Administration seeking authorization to expand the use of its Edison® Histotripsy System to include the destruction of kidney (renal) tumors. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260511268688/en/ HistoSonics Edison® Histotripsy System This milestone marks a significant step forward in the company’s mission to transform the treatment of solid tumors with a completely non-invasive technology that harnesses focused ultrasound to mechanically liquefy and destroy targeted tissue, reducing the risk of many complications and side effects associated with surgery, radiation, and other common therapies. “This submission is an important milestone in expanding histotripsy beyond the liver and into the kidney, an area where patients and physicians are s
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