Drugs / fidanacogene elaparvovec
fidanacogene elaparvovec
also known as
PF-06838435 · SPK-9001 · AAV8-hFIX19 · BEQVEZ · Durveqtix · Fidanacogen elaparvovec · Raav-spark100-hfix-padua · Spark 101 · +3 more
Gene therapy (AAV / viral vector) targets F9 via restoration
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed hemophilia B leyden 2 milestones | |||||
| Filed | US (FDA) | — | hemophilia B leyden | 2023-06-27 | “SPK-9001 for the treatment of Hemophilia B (marketing applications currently under review by FDA and EMA)” 4dmoleculartherapeutics.com ↗ “(FDA) has accepted the company’s Biologics License Application (BLA) for fidanacogene elaparvovec” pfizer.com ↗ |
| Filed | EU (EMA) | — | hemophilia B leyden | 2023-06-27 | “In parallel, the European marketing authorization application (MAA) for fidanacogene elaparvovec has also been accepted and is under review by the European Medicines Agency (EMA).” pfizer.com ↗ “SPK-9001 for the treatment of Hemophilia B (marketing applications currently under review by FDA and EMA)” 4dmoleculartherapeutics.com ↗ |
| Withdrawn Indication not stated 2 milestones | |||||
| Withdrawn | EU (EMA) | Beqvez (previously Durveqtix) | — | 2025-05-15 | europa.eu ↗ |
| Approved · conditional | EU (EMA) | Beqvez (previously Durveqtix) | — | 2024-07-24 | europa.eu ↗ |
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT01620801 | Oct 2012 → Mar 2016 | hemophilia B | Spark Therapeutics, Inc. | Terminated | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT03307980 | Jun 2017 → May 2029 expected | hemophilia B | Pfizer | Active not recruiting | No outcome recorded |
| Phase 2 | NCT02484092 | Nov 2015 → Apr 2019 | hemophilia B | Pfizer | Completed | No outcome recorded |
| Phase 31 trial · 1 met primary | ||||||
| Phase 3 | NCT03861273 | Jul 2019 → Nov 2022 overdue | hemophilia B | Pfizer | Active not recruiting | Met primary |
News releases announcing trial results or a regulatory action · 15
| Date | Issuer | Release |
|---|---|---|
| 2024-07-25 | Pfizer | Regulatory European Commission Approves Pfizer’s DURVEQTIX® (fidanacogene elaparvovec), a One-Time Gene Therapy for Adults with Hemophilia B pfizer.com ↗
The EC approval follows recent regulatory approvals by the U.S. Food and Drug Administration (FDA) and Health Canada, where it is marketed as BEQVEZ TM . |
| 2024-04-26 | Pfizer | Regulatory U.S. FDA Approves Pfizer’s BEQVEZ™ (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B pfizer.com ↗
Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. |
| 2023-06-27 | Pfizer | Regulatory FDA Accepts Pfizer’s Application for Hemophilia B Gene Therapy Fidanacogene Elaparvovec pfizer.com ↗
Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for fidanacogene elaparvovec for the treatment of adults with hemophilia B. |
| 2022-12-29 | Pfizer | Results Pfizer Announces Positive Top-Line Results from Phase 3 Study of Hemophilia B Gene Therapy Candidate pfizer.com ↗
The BENEGENE-2 study met its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds post-fidanacogene elaparvovec infusion versus prophylaxis regimen with Factor IX (FIX), administered as part of usual care. |
| 2018-05-21 | Pfizer | Results Spark Therapeutics and Pfizer Announce Data from 15 Participants with Hemophilia B Showing Persistent and Sustained Factor IX Levels with No Serious Adverse Events pfizer.com ↗
None of the 15 participants experienced serious adverse events, and there were no thrombotic events or factor IX inhibitors, as of the May 7, 2018 data cutoff. |
| 2017-12-11 | Pfizer | Results Spark Therapeutics and Pfizer Announce Longer-term Preliminary Data Showing Consistent and Sustained Factor IX Levels in Hemophilia B at 59th American Society of Hematology (ASH) Annual Meeting and Exposition pfizer.com ↗
We believe these longer-term data are meeting critical goals of our hemophilia programs |
| 2017-12-06 | Pfizer | Results Spark Therapeutics and Pfizer Announce Publication in The New England Journal of Medicine of Interim Data from Phase 1/2 Clinical Trial of Investigational Gene Therapy for Hemophilia B pfizer.com ↗
The annualized bleeding rate (ABR) was reduced 97 percent, from a mean rate of 11.1 events per year before vector administration to 0.4 events per year after vector administration (p=0.02), while factor IX concentrate use was reduced 99 percent (p=0.004). |
| 2017-07-10 | Pfizer | Results Spark Therapeutics Presents Updated Interim Hemophilia B Data Supporting Consistent and Sustained Response at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress pfizer.com ↗
Data demonstrate a 99-percent reduction in annualized infusion rate (AIR) and a 96-percent reduction in annualized bleeding rate (ABR) in 10 participants as of June 5, 2017 |
| 2017-06-04 | Pfizer | Results Spark Therapeutics Presents Updated Preliminary Data From Hemophilia B Phase 1/2 Trial Suggesting Consistent And Sustained Levels Of Factor IX Activity At The Hemostasis And Thrombosis Research Society (HTRS) 2017 Scientific Symposium pfizer.com ↗
Spark Therapeutics (NASDAQ:ONCE) announced updated preliminary data today from 10 infused participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for hemophilia B. |
| 2017-03-01 | Pfizer | Regulatory Spark Therapeutics and Pfizer Announce that SPK-9001, an Investigational Hemophilia B Medicine, has been Granted Access to the PRIority MEdicines (PRIME) Program by the European Medicines Agency pfizer.com ↗
Spark Therapeutics (NASDAQ:ONCE) and Pfizer Inc. (NYSE:PFE) announced today that SPK-9001, the lead investigational candidate in the companies' SPK-FIX program has been granted support through the European Medicines Agency (EMA) PRIority MEdicines (PRIME) program. |
| 2016-12-03 | Pfizer | Results Spark Therapeutics and Pfizer Present Updated Data from Hemophilia B Phase 1/2 Trial Suggesting Consistent and Sustained Levels of Factor IX Activity at Annual ASH Meeting pfizer.com ↗
The first participant to reach one year in the study who has been followed for 52 weeks post-infusion of SPK-9001, has reduced to zero his number of intravenous factor IX infusions without having any bleeds. |
| 2016-11-03 | Pfizer | Results Spark Therapeutics and Pfizer Announce Updated Data from Hemophilia B Phase 1/2 Trial Suggesting Sustained Therapeutic Levels of Factor IX Activity pfizer.com ↗
Four of the seven participants had reached greater than 12 weeks post-vector administration at the time of abstract submission; those four participants experienced consistent and sustained factor IX activity levels, with a mean greater than 30% of normal, with no sustained elevation in liver enzyme levels. |
| 2016-07-25 | Pfizer | Results Spark Therapeutics and Pfizer Announce Updated Data from First Cohort in Hemophilia B Phase 1/2 Trial Demonstrating Consistent, Sustained Therapeutic Levels of Factor IX Activity pfizer.com ↗
Across the four subjects, average steady-state factor IX activity levels are 31.8% ±6.9% (range 20% - 44%) of normal, determined by averaging levels beginning at 8 weeks post vector administration through follow up over 12-31 weeks. |
| 2016-07-21 | Pfizer | Regulatory Spark Therapeutics and Pfizer Announce Receipt of FDA Breakthrough Therapy Designation for SPK-9001 for the Treatment of Hemophilia B pfizer.com ↗
the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to SPK-9001, the lead investigational candidate in the companies’ SPK-FIX program, in development for the treatment of hemophilia B. |
| 2016-05-19 | Pfizer | Results Spark Therapeutics and Pfizer Announce Data from Initial Subjects in Hemophilia B Trial Demonstrating Consistent Therapeutic Levels of Factor IX Expression pfizer.com ↗
Data available today demonstrate that the first three subjects enrolled in the study experienced AAV-mediated Factor IX activity levels following one administration of SPK-9001 at the initial dose level (5 x 1011 vg/kg) studied in the trial. |
All press releases naming this drug 16 releases
Evidence & citations 16 cited values
Every value below carries the sentence it was read from. 6 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | fidanacogene elaparvovec | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03861273 ↗ |
| Known as | AAV8-hFIX19 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01620801 ↗ |
| Known as | BEQVEZ | “Fidanacogene elaparvovec (BEQVEZ™), an adeno-associated virus-based gene therapy approved for the treatment of hemophilia B” PMID 40750723 ↗ Aug 2025 |
| Known as | Durveqtix | ChEMBL registry synonym — accepted as the source's own label CHEMBL3990034 ↗ |
| Known as | Fidanacogen elaparvovec | ChEMBL registry synonym — accepted as the source's own label CHEMBL3990034 ↗ |
| Known as | PF-06838435 | “Gene Therapy, Open-label, Dose-escalation Study of PF-06838435 (SPK-9001) [Adeno-associated Viral Vector With Human Factor IX Gene] in Subjects With Hemophilia B” NCT02484092 ↗ |
| Known as | Raav-spark100-hfix-padua | ChEMBL registry synonym — accepted as the source's own label CHEMBL3990034 ↗ |
| Known as | Spark 101 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3990034 ↗ |
| Known as | SPK 101 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3990034 ↗ |
| Known as | SPK-1001 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3990034 ↗ |
| Known as | SPK-9001 | “PF-06838435 (formerly SPK-9001)” NCT03307980 ↗ |
| Known as | SPK-FIX Padua | ChEMBL registry synonym — accepted as the source's own label CHEMBL3990034 ↗ |
| Action | Restore | “to deliver the human factor IX (hFIX) gene to the liver cells where FIX is normally made” NCT02484092 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “This study plans to use a virus called adeno-associated virus (AAV), which in nature causes no disease, and can be engineered to deliver the human FIX gene (AAV8-hFIX19 vector)...” NCT01620801 ↗ |
| Route | Intravenous | “The vector will be injected once into a peripheral vein of each subject” NCT01620801 ↗ |
| Target | F9 | “to deliver the human factor IX (hFIX) gene to the liver cells where FIX is normally made” NCT02484092 ↗ |