Drugs / giroctocogene fitelparvovec
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold | US (FDA) | — | hemophilia B leyden | 2021-12-12 | “Food and Drug Administration (FDA) informed Pfizer that this trial has been placed on clinical hold while the protocol amendment and associated documents are reviewed.” pfizer.com ↗ |
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 21 trial | ||||||
| Phase 2 | NCT03061201 | Jun 2017 → Jul 2024 | hemophilia A | Pfizer | Completed | No outcome recorded |
| Phase 31 trial · 1 met primary | ||||||
| Phase 3 | NCT04370054 | Aug 2020 → Jun 2024 overdue | hemophilia A | Pfizer | Active not recruiting | Met primary |
News releases announcing trial results or a regulatory action · 11
| Date | Issuer | Release |
|---|---|---|
| 2025-11-03 | Gritgen Therapeutics Co., Ltd. | Results 2025-11-03 2025血栓与止血学术会议｜王华芳教授：血友病A基因治疗的疗效和安全性良好，临床应用前景可期 gritgen.com ↗ |
| 2024-07-24 | Pfizer | Results Pfizer Announces Positive Topline Results From Phase 3 Study of Hemophilia A Gene Therapy Candidate pfizer.com ↗
Pfizer Inc. (NYSE: PFE) today announced positive topline results from the Phase 3 AFFINE study ( NCT04370054 ) evaluating giroctocogene fitelparvovec, an investigational gene therapy for the treatment of adults with moderately severe to severe hemophilia A. |
| 2021-12-12 | Pfizer | Results Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Bleeding Control in Highest Dose Cohort Through Two Years Following Hemophilia A Gene Therapy pfizer.com ↗
At 104 weeks, the five patients in the highest dose 3e13 vg/kg cohort had mean factor VIII (FVIII) activity of 25.4% via chromogenic clotting assay. |
| 2020-12-07 | Pfizer | Results Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Factor VIII Activity Levels in 3x10¹³ VG/KG Cohort Through One Year Following Hemophilia A Gene Therapy pfizer.com ↗
All five patients in the high dose 3 x 10 13 vg/kg cohort have had at least one year of follow-up and showed sustained factor VIII (FVIII) activity levels, with a group median FVIII activity of 56.9% and a group geometric mean FVIII activity of 70.4% via chromogenic assay from week 9 to 52. |
| 2020-06-18 | Pfizer | Results Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Factor VIII Activity Levels and No Bleeding Events or Factor Usage in 3e13 vg/kg Cohort Following giroctocogene fitelparvovec (SB-525) Gene Therapy pfizer.com ↗
No patients experienced bleeding events or required FVIII infusions. |
| 2019-12-07 | Pfizer | Results Sangamo and Pfizer Announce Updated Phase 1/2 Results Showing Sustained Increased Factor VIII Activity Through 44 Weeks Following SB-525 Gene Therapy Treatment pfizer.com ↗
The data showed that SB-525 was generally well tolerated and demonstrated sustained increased Factor VIII (FVIII) levels following treatment with SB-525 through to 44 weeks, the extent of follow-up for the longest treated patient in the 3e13 vg/kg dose cohort. |
| 2019-07-05 | Pfizer | Results Sangamo and Pfizer Announce Updated Phase 1/2 Results for SB-525 Investigational Hemophilia A Gene Therapy Showing Sustained Increased Factor VIII Levels pfizer.com ↗
The data showed that SB-525 was generally well-tolerated and demonstrated a dose-dependent increase in Factor VIII (FVIII) activity levels. |
| 2019-04-02 | Pfizer | Results Sangamo and Pfizer Announce Phase 1/2 Interim Data for Investigational Hemophilia A Gene Therapy pfizer.com ↗
BRISBANE, Calif. & Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, and Pfizer, Inc. (NYSE: PFE) today announced interim data from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. |
| 2018-08-08 | Pfizer | Results SANGAMO ANNOUNCES POSITIVE PRELIMINARY DATA FROM THE PHASE 1/2 ALTA STUDY EVALUATING SB-525 GENE THERAPY FOR HEMOPHILIA A pfizer.com ↗
The fifth patient in the study, the first at the third dose level, was treated in June and has achieved therapeutic Factor VIII activity levels.* |
| 2017-06-07 | Pfizer | Regulatory Sangamo Therapeutics And Pfizer Announce That Sb-525 Investigational Hemophilia A Gene Therapy Receives Orphan Medicinal Product Designation From The European Medicines Agency pfizer.com ↗
Sangamo Therapeutics, Inc. (NASDAQ: SGMO) and Pfizer Inc. (NYSE: PFE) announced today that the European Medicines Agency (EMA) has granted orphan medicinal product designation (OMPD) to SB-525, a clinical stage cDNA gene therapy candidate for hemophilia A. |
| 2017-05-16 | Pfizer | Regulatory Sangamo Receives Fast Track Designation From The Fda For Sb-525 Investigational Hemophilia A Gene Therapy pfizer.com ↗ |
All press releases naming this drug 12 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | giroctocogene fitelparvovec | “PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy)” NCT04370054 ↗ |
| Known as | Giroctocogen fitelparvovec | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298194 ↗ |
| Known as | PF-07055480 | “PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy)” NCT04370054 ↗1“Single dose of investigational product SB-525 (PF-07055480)” NCT03061201 ↗ |
| Known as | SB-525 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03061201 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Recombinant AAV2/6 Human Factor VIII Gene Therapy” NCT04370054 ↗ |
| Route | Intravenous | “Single IV infusion” NCT04370054 ↗ |
| Target | F8 | “Coagulation factor VIII exogenous gene” CHEMBL4298194 ↗ |