Drugs / AAV5-hFIXco-Padua
AAV5-hFIXco-Padua
Regulatory milestones approvals, filings & regulatory actions · 3 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed hemophilia B leyden 2 milestones | |||||
| Filed | US (FDA) | — | hemophilia B leyden | 2022-05-24 | “the Biologics License Application (BLA) submitted by CSL Behring for etranacogene dezaparvovec, an investigational gene therapy for the treatment of adults with hemophilia B.” gcs-web.com ↗ “the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA), for priority review, for etranacogene dezaparvovec” csl.com ↗ “FDA has accepted for priority review the Biologics License Application (BLA) submitted by CSL Behring for etranacogene dezaparvovec” gcs-web.com ↗ |
| Filed | EU (EMA) | — | hemophilia B leyden | 2022-03-28 | “the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for etranacogene dezaparvovec (EtranaDez)” csl.com ↗ “Partner CSL Behring submitted Marketing Authorization Application (MAA) in Europe for etranacogene dezaparvovec in hemophilia B” gcs-web.com ↗ “MAA validated by the European Medicines Agency (EMA) and granted accelerated assessment” gcs-web.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved · conditional | EU (EMA) | Hemgenix | — | 2023-02-20 | europa.eu ↗ |
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 21 trial · 1 met primary | ||||||
| Phase 2 | NCT03489291 | Jul → Oct 2018 | hemophilia B | CSL Behring | Completed | Met primary |
| Phase 31 trial · 1 met primary | ||||||
| Phase 3 | NCT03569891 | Jun 2018 → Sep 2021 | hemophilia B | CSL Behring | Completed | Met primary |
News releases announcing trial results or a regulatory action · 31
| Date | Issuer | Release |
|---|---|---|
| 2025-12-07 | CSL Behring | Results Data Published in the New England Journal of Medicine Confirm the Long-term Durability and Safety of HEMGENIX® (etranacogene dezaparvovec-drlb) Over Five Years csl.com ↗ |
| 2025-10-06 | CSL Behring | Regulatory CSL Behring Signs Letter of Intent with pan-Canadian Pharmaceutical Alliance (pCPA) for Public Reimbursement of HEMGENIX® (etranacogene dezaparvovec), the First Gene Therapy for Hemophilia B csl.com ↗ |
| 2025-02-07 | CSL Behring | Results CSL Behring's Gene Therapy HEMGENIX® (etranacogene dezaparvovec-drlb) Four Years Post-Infusion Data Continue to Show Sustained Efficacy and Safety in Adults with Hemophilia B csl.com ↗
Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced the four-year results from the pivotal HOPE-B study confirming the long-term durability and safety of a one-time infusion of HEMGENIX ® (etranacogene dezaparvovec-drlb) for adults living with hemophilia B. |
| 2024-01-15 | CSL Behring | Regulatory Swissmedic Authorises CSL's HEMGENIX® (etranacogene dezaparvovec) as First Gene Therapy for Hemophilia B csl.com ↗
CSL (ASX: CSL) today announced that Swissmedic has authorised HEMGENIX ® (etranacogene dezaparvovec), the first and currently only gene therapy for the treatment of male adults aged >18 years with severe or moderately severe hemophilia B (congenital Factor IX deficiency). |
| 2023-12-11 | CSL Behring | Results CSL Behring's HEMGENIX® (etranacogene dezaparvovec-drlb) Demonstrates at Three Years Post-Treatment Long-Term Durability, Safety and Greater Bleed Protection Versus Prophylactic Treatment in People Living with Hemophilia B csl.com ↗
Global biotechnology leader CSL (ASX: CSL; USOTC: CSLLY) today announced the three-year results from the pivotal HOPE-B study confirming continued long-term durability and safety of HEMGENIX ® (etranacogene dezaparvovec-drlb) following a one-time infusion in people living with hemophilia B. |
| 2023-10-26 | CSL Behring | Regulatory Health Canada Authorizes CSL's HEMGENIX® (etranacogene dezaparvovec) as First Gene Therapy for Hemophilia B csl.com ↗
CSL (ASX: CSL) today announced that Health Canada has authorized HEMGENIX® (etranacogene dezaparvovec), the first and only gene therapy for the treatment of hemophilia B. |
| 2023-02-23 | CSL Behring | Results CSL's HOPE-B Data Published in the New England Journal of Medicine, Demonstrating Efficacy and Durability of HEMGENIX® (etranacogene dezaparvovec-drlb) csl.com ↗
Increased factor IX activity was also apparent from week 3 and maintained over 18 months. |
| 2023-02-21 | UniQure Biopharma B.V. | Regulatory uniQure announces the European Commission approval of the first gene therapy for adults with hemophilia B gcs-web.com ↗
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that its partner, global biotechnology leader CSL (ASX: CSL), has received conditional marketing authorization (CMA) from the European Commission for HEMGENIX ® (etranacogene dezaparvovec), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B. |
| 2023-02-20 | CSL Behring | Regulatory First Gene Therapy for Hemophilia B, CSL's HEMGENIX®, Approved by the European Commission csl.com ↗
Global biotechnology leader CSL (ASX: CSL) today announced that the European Commission has granted conditional marketing authorization (CMA) for HEMGENIX ® (etranacogene dezaparvovec), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B (congenital Factor IX deficiency) in adults without a history of Factor IX inhibitors. |
| 2022-12-16 | UniQure Biopharma B.V. | Regulatory uniQure announces positive CHMP opinion for etranacogene dezaparvovec – gene therapy for adults with hemophilia B gcs-web.com ↗ |
| 2022-12-16 | CSL Behring | Regulatory CSL Receives Positive CHMP Opinion for Etranacogene Dezaparvovec – Gene Therapy for Adults with Hemophilia B csl.com ↗
CSL (ASX: CSL) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) of etranacogene dezaparvovec. |
| 2022-12-10 | CSL Behring | Results CSL's Novel Gene Therapy HEMGENIX® (etranacogene dezaparvovec-drlb) Demonstrates Durable Protection and Sustained Factor IX Activity Levels for People Living with Hemophilia B at 24-Months Post-Treatment csl.com ↗ |
| 2022-11-22 | UniQure Biopharma B.V. | Regulatory uniQure announces FDA approval of first gene therapy for adults with hemophilia B gcs-web.com ↗
CSL (ASX: CSL), has received approval from the U.S. Food and Drug Administration (FDA) for HEMGENIX ® (etranacogene dezaparvovec-drlb), a one-time gene therapy for the treatment of adults 18 years of age and older living with hemophilia B. |
| 2022-11-22 | CSL Behring | Regulatory U.S. Food and Drug Administration approves CSL's HEMGENIX® (etranacogene dezaparvovec-drlb), the first gene therapy for hemophilia B csl.com ↗
Global biotechnology leader CSL (ASX: CSL) today announced that the U.S. Food and Drug Administration (FDA) approved HEMGENIX ® (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with hemophilia B. |
| 2022-11-02 | CSL Behring | Regulatory Next-Generation mRNA, Gene Therapy, Plasma Products, Monoclonal Antibodies, and Recent Acquisitions and Collaborations Highlight CSL R&D Day 2022 csl.com ↗
Etranacogene dezaparvovec (also known as CSL222), an investigational gene therapy for the treatment of adults with haemophilia B has been accepted for priority review by the United States (US) Food and Drug Administration (FDA) and standard review by the European Medicines Agency (EMA). |
| 2022-09-08 | CSL Behring | Regulatory Orphan Drug Designation granted for etranacogene dezaparvovec, CSL’s gene therapy for haemophilia B, by the Therapeutics Goods Administration csl.com ↗
Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) announced that the Therapeutic Goods Administration (TGA) has granted Orphan Drug Designation for etranacogene dezaparvovec, a late-stage gene therapy candidate developed for haemophilia B. |
| 2022-05-24 | UniQure Biopharma B.V. | Regulatory uniQure Announces FDA Acceptance of Biologics License Application for Etranacogene Dezaparvovec under Priority Review gcs-web.com ↗ |
| 2022-05-24 | CSL Behring | Regulatory FDA Accepts CSL Behring's Biologics License Application for Etranacogene Dezaparvovec for Priority Review csl.com ↗
FDA's acceptance of the etranacogene dezaparvovec BLA for priority review is another pivotal milestone in CSL Behring's commitment to deliver innovative treatment options that address unmet needs for people living with rare diseases |
| 2022-05-02 | UniQure Biopharma B.V. | Regulatory uniQure Announces First Quarter 2022 Financial Results and Highlights Recent Company Progress gcs-web.com ↗
Partner CSL Behring submitted Marketing Authorization Application (MAA) in Europe for etranacogene dezaparvovec in hemophilia B; MAA validated by the European Medicines Agency (EMA) and granted accelerated assessment |
| 2022-03-28 | CSL Behring | Regulatory European Medicines Agency Commences Review of Novel Gene Therapy Candidate Etranacogene Dezaparvovec for People with Hemophilia B csl.com ↗
CSL Behring today announced that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for etranacogene dezaparvovec (EtranaDez) under its accelerated assessment procedure. |
| 2022-02-04 | CSL Behring | Results Final Analysis of Pivotal HOPE-B Study Demonstrates Durable and Sustained Therapeutic Effect of Etranacogene Dezaparvovec Gene Therapy in Hemophilia B - Data Presented at EAHAD 2022 csl.com ↗
Global biotherapeutics leader CSL Behring today announced positive long-term results from the Phase 3 HOPE-B clinical trial evaluating etranacogene dezaparvovec (EtranaDez), an investigational adeno-associated virus five (AAV5)-based gene therapy for people living with hemophilia B, a life-threatening bleeding disorder. |
| 2021-05-13 | UniQure Biopharma B.V. | Results uniQure Presents HOPE-B Clinical Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-existing Antibodies to AAV5 Vector gcs-web.com ↗ |
| 2021-04-26 | UniQure Biopharma B.V. | Regulatory uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program gcs-web.com ↗ |
| 2020-12-21 | UniQure Biopharma B.V. | Regulatory uniQure Announces Clinical Update on Hemophilia B Gene Therapy Program gcs-web.com ↗
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that its hemophilia B gene therapy program, including the pivotal, Phase III HOPE-B study, has been placed on clinical hold by the U.S. Food and Drug Administration (FDA). |
| 2019-10-31 | UniQure Biopharma B.V. | Results uniQure Announces Publication of Positive Interim Data from Phase IIb Clinical Trial of Etranacogene Dezaparvovec gcs-web.com ↗
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced the publication of 26-week interim data from the ongoing Phase IIb clinical trial of etranacogene dezaparvovec |
| 2019-07-06 | UniQure Biopharma B.V. | Results uniQure Announces 36 Weeks of Follow-Up Data from Phase IIb Study of AMT-061 and Long-Term Follow-Up Data for AMT-060 in Patients with Hemophilia B gcs-web.com ↗
FIX Activity Up to 54% of Normal, with Mean of 45% of Normal, at 36 Weeks After Administration of AMT-061 in Phase IIb Study |
| 2019-05-10 | UniQure Biopharma B.V. | Results uniQure Announces Updated Clinical Data from Phase IIb Study of AMT-061 in Patients with Hemophilia B gcs-web.com ↗
Increases in FIX Activity Sustained at up to 57% of Normal, with Mean FIX of 47% of Normal at Six Months After Administration |
| 2019-02-08 | UniQure Biopharma B.V. | Results uniQure Announces Updated Clinical Data from Phase IIb Study of AMT-061 in Patients with Hemophilia B gcs-web.com ↗
Increases in FIX Activity Sustained at up to 51% of Normal, with Mean FIX of 38% of Normal at Twelve Weeks After Administration |
| 2018-11-15 | UniQure Biopharma B.V. | Results uniQure Announces Initial Topline Data from Dose-Confirmation Study of AMT-061 in Patients with Hemophilia B gcs-web.com ↗
These data show that therapeutic levels of Factor IX (FIX) activity have been achieved and sustained in all three patients at six weeks after a single administration of AMT-061. |
| 2017-12-09 | UniQure Biopharma B.V. | Results uniQure Announces Presentation of Non-Clinical Data of AMT-061 at the 59th American Society of Hematology (ASH) Annual Meeting gcs-web.com ↗
Animals receiving the same dose of AMT-060 and AMT-061 showed comparable levels of hFIX protein, but the animals receiving AMT-061 demonstrated approximately 6.5-fold higher FIX activity compared to those receiving AMT-060. |
| 2017-11-01 | UniQure Biopharma B.V. | Results uniQure Announces Presentations at the 59th American Society of Hematology (ASH) Annual Meeting gcs-web.com ↗
Data from the study demonstrated a strong correlation between dose and human FIX (hFIX) expression levels, as well as biological activity of the expressed hFIX protein. |
All press releases naming this drug 42 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AAV5-hFIXco-Padua | “The IMP is identified as AAV5-hFIXco-Padua (AMT- 061).” NCT03489291 ↗ |
| Known as | AMT-061 | “Etranacogene dezaparvovec (AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing a codon-optimized Padua variant human factor IX (FIX) transgene...” PMID 36490302 ↗ Oct 2023 |
| Known as | etranacogene dezaparvovec | “Etranacogene dezaparvovec (AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing a codon-optimized Padua variant human factor IX (FIX) transgene...” PMID 36490302 ↗ Oct 2023 |
| Modality | Gene therapy (AAV / viral vector) | “recombinant AAV5 vector” PMID 31698454 ↗ Nov 2019 |
| Route | Intravenous | “Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)” NCT03489291 ↗ |