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
Bregal Milestone III Closes at its Increased Hard Cap of €915 Million7.7.2026 13:00:00 CEST | Press release
Oversubscribed fundraise with global institutional backing Bregal Milestone (“Milestone” or the “Firm”), a leading European software private equity firm, today announced the final close of its third fund, Bregal Milestone III (“Fund III” or the “Fund”), at its increased hard cap of €915 million. The Fund was oversubscribed and marks a significant broadening of the Firm’s global investor base. Completed in eight months from first close and achieved against a challenging fundraising environment, the result reflects the strength of Milestone’s AI-centric approach and demonstrable track record. Jan Bruennler, Philippe Lautenberg and Cyrus Shey, Co-Founders and Managing Partners at Bregal Milestone, commented: “Bregal Milestone III reflects strong conviction in our approach to investing in mission-critical European software, AI and cybersecurity businesses and helping them scale. We are thankful for the support and confidence shown by our limited partners, both longstanding and new, and rem
HTEC and Embotech Partner to Scale Deployment of Autonomous Driving Solutions for Industrial Logistics7.7.2026 12:39:00 CEST | Press release
HTEC, a global technology and AI engineering company headquartered in Silicon Valley, and Embotech, a global leader in safety-certified Level 4 autonomous driving solutions for industrial logistics, today announced a strategic partnership to scale deployment of Embotech's autonomous driving solutions across vehicle platforms, customer programmes and industrial sites. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260707870943/en/ HTEC, a global technology and AI engineering company headquartered in Silicon Valley, and Embotech, a global leader in safety-certified Level 4 autonomous driving solutions for industrial logistics, today announced a strategic partnership to scale deployment of Embotech's autonomous driving solutions across vehicle platforms, customer programmes and industrial sites. The partnership comes as Embotech’s proven autonomous driving solutions are being adopted across a growing number of vehicle platforms
Compass Pathways Announces Six-Month Data from Second Phase 3 Trial Confirming Rapid and Durable Profile7.7.2026 12:30:00 CEST | Press release
New 6-month data supports COMP360’s potential to establish a new standard of care in TRDData from positive COMP006 Phase 3 trial demonstrates durable benefit through at least 6 months further validating COMP360’s differentiated profile in highly chronic TRDAcross two highly statistically significant positive Phase 3 trials, COMP360 is the first classic psychedelic¹ to consistently demonstrate rapid onset, significant durability and reproducibility of effect in TRD, one of psychiatry’s most difficult conditions in which to demonstrate efficacyCOMP360 continues to demonstrate a generally well-tolerated and safe profile with no new safety findingsRolling NDA submission and initial review underway and final submission expected to be completed in Q4Commercial launch-readiness on track for end of 2026, with launch expected in first half of 2027Compass continues to progress late-stage PTSD program, where millions of patients are urgently in need of new, effective optionsCompass to host a webi
Private Department of Sheikh Mohammed bin Khalid Al Nahyan Invests in MidOcean Energy and Forms Strategic Partnership with EIG7.7.2026 12:16:00 CEST | Press release
The Private Department of Sheikh Mohammed bin Khalid Al Nahyan (the “Private Department”) has today committed to invest $1.13bn in MidOcean Energy (“MidOcean” or the “Company”), a liquefied natural gas (LNG) company formed and managed by EIG. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260707259928/en/ In parallel with the investment, the Private Department and EIG have established a strategic partnership focused on capital aggregation, investment origination, and the development of institutional investment opportunities across the United Arab Emirates and selected regional markets. The Investment in MidOcean represents the Private Department’s entry into the global LNG sector and marks the launch of a broader strategic relationship with EIG, a global energy and infrastructure investment firm. Through this partnership, the parties intend to collaborate on future investment opportunities across the energy and related infra
Infobip Research Reveals Businesses Scaling AI-Powered Defenses to Counter Surge in Automated Fraud7.7.2026 11:55:00 CEST | Press release
Fraudsters are leveraging AI to automate and personalize attacks, but enterprises are fighting back New research from global AI-first cloud communications platform Infobip reveals a growing surge in AI-powered fraud and enterprise defenses. Analyzing billions of interactions globally, Infobip’s 2026 Fraud & Security Report highlights a year of dramatic contrasts. There have been record volumes of blocked fraudulent traffic alongside the rapid scaling of intelligent, AI-powered defenses. The report reveals that while fraudsters are using AI to scale and personalize harmful messaging, leading to a 77% increase in detected threats, businesses are responding in kind. Adoption of AI-powered fraud detection grew by 71% year-on-year, while pattern-based detection increased by 105%, underscoring a shift toward adaptive security. The report demonstrates the impact of AI-first security. Leading UK-based clothing brand NEXT is leveraging Infobip Signals, an AI and machine learning-powered solutio
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