Drugs / valoctocogene roxaparvovec
Regulatory milestones approvals, filings & regulatory actions · 6 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed hemophilia B leyden 5 milestones | |||||
| Filed | US (FDA) | — | hemophilia B leyden | 2022-09-29 | “resubmitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec” biomarin.com ↗ |
| Filed | EU (EMA) | — | hemophilia B leyden | 2021-06-25 | “resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with...” biomarin.com ↗ “the European Medicines Agency validated a Marketing Authorization Application in the EU for valoctocogene roxaparvovec” biomarin.com ↗ “The European Medicines Agency (EMA) validated BioMarin's MAA for valoctocogene roxaparvovec resulting” biomarin.com ↗ “The European Medicines Agency (EMA) validated BioMarin's MAA for valoctocogene roxaparvovec:” biomarin.com ↗ |
| Rejected (CRL) | US (FDA) | — | hemophilia B leyden | 2020-08-18 | “the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to the Company's Biologics License Application (BLA) for valoctocogene roxaparvovec gene...” biomarin.com ↗ “following the August 18, 2020 Complete Response Letter to the Company's Biologics License Application for valoctocogene roxaparvovec gene therapy for severe hemophilia A” biomarin.com ↗ “The resubmission incorporates the Company's response to the FDA Complete Response (CR) Letter for valoctocogene roxaparvovec gene therapy issued on August 18, 2020” biomarin.com ↗ |
| Filed | US (FDA) | — | hemophilia B leyden | 2019-12-23 | “company submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for...” biomarin.com ↗ |
| Filed | EU (EMA) | — | hemophilia B leyden | 2019-11-21 | “company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec, for...” biomarin.com ↗ “European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults...” biomarin.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved · conditional | EU (EMA) | Roctavian | — | 2022-08-24 | europa.eu ↗ |
Trials 7
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/23 trials · 1 met primary | ||||||
| Phase 1/2 | NCT04684940 | Dec 2020 → Apr 2026 | hemophilia A | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 1/2 | NCT03520712 | Apr 2018 → Aug 2024 | hemophilia A | BioMarin Pharmaceutical | Terminated | No outcome recorded |
| Phase 1/2 | NCT02576795 | Sep 2015 → Feb 2024 | severe hemophilia A | BioMarin Pharmaceutical | Completed | Met primary |
| Phase 34 trials · 2 met primary | ||||||
| Phase 3 | NCT06224907 | Dec 2023 → Apr 2025 overdue | hemophilia A | BioMarin Pharmaceutical | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04323098 | Dec 2020 → Jan 2023 | hemophilia A | BioMarin Pharmaceutical | Completed | Met primary |
| Phase 3 | NCT03392974 | Mar 2018 → May 2019 | hemophilia A | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 3 | NCT03370913 | Dec 2017 → Nov 2020 | hemophilia A | BioMarin Pharmaceutical | Completed | Met primary |
News releases announcing trial results or a regulatory action · 43
| Date | Issuer | Release |
|---|---|---|
| 2025-06-24 | BioMarin Pharmaceutical | Results BioMarin Presents Five-Year Phase 3 Results Reinforcing Long-Term Efficacy and Safety of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) at International Society on Thrombosis and Haemostasis 2025 Congress biomarin.com ↗
The Phase 3 GENEr8-1 trial demonstrated that durable bleed control and sustained factor VIII (FVIII) expression were maintained five years after treatment with ROCTAVIAN. |
| 2024-06-07 | BioMarin Pharmaceutical | Results BioMarin Presents New Phase 3, Four-Year Data Underscoring Long-Term Safety and Efficacy of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) at International Society on Thrombosis and Haemostasis 2024 Congress biomarin.com ↗
The Phase 3 GENEr8-1 trial demonstrated that durable bleed control and sustained factor VIII (FVIII) expression were maintained four years after treatment with ROCTAVIAN, with FVIII activity near stable compared with results reported previously and no new safety signals observed. |
| 2024-02-06 | BioMarin Pharmaceutical | Results BioMarin to Present ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) Data Highlighting Long-Term Durability at 2024 European Association for Haemophilia and Allied Disorders (EAHAD) Congress biomarin.com ↗
The majority of participants maintained hemostasis, with only two of the seven participants in the study returning to regular prophylaxis, and no new safety signals emerged. |
| 2023-06-29 | BioMarin Pharmaceutical | Regulatory U.S. Food and Drug Administration Approves BioMarin’s ROCTAVIAN™ (valoctocogene roxaparvovec-rvox), the First and Only Gene Therapy for Adults with Severe Hemophilia A biomarin.com ↗ |
| 2023-03-06 | BioMarin Pharmaceutical | Regulatory BioMarin Provides Update on FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) Gene Therapy for Adults with Severe Hemophilia A biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that it received notice this afternoon from the U.S. Food and Drug Administration (FDA) that the agency has extended review of the company's Biologics License Application (BLA) for ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy for adults with severe hemophilia A. |
| 2023-01-08 | BioMarin Pharmaceutical | Results BioMarin Announces Stable and Durable Annualized Bleed Control for ROCTAVIAN™ in Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A; 134-Participant Study Met All Primary and Secondary Efficacy Endpoints at 3-Year Analysis biomarin.com ↗
Mean Annualized Bleed Rate Reduced by 80% from Baseline and Factor VIII Usage Reduced by 94% in Year 3 Compared to Baseline |
| 2022-11-23 | BioMarin Pharmaceutical | Regulatory BioMarin Announces Advancements in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A biomarin.com ↗
The Company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review. |
| 2022-11-07 | BioMarin Pharmaceutical | Regulatory BioMarin Announces Incremental Progress on Biologics License Application (BLA) Review for Valoctocogene Roxaparvovec AAV Gene Therapy for Adults with Severe Hemophilia A Program biomarin.com ↗
As part of their review of the BLA, the FDA has scheduled its Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility, located in Novato, CA. |
| 2022-10-12 | BioMarin Pharmaceutical | Regulatory FDA Accepts BioMarin’s Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Adults with Severe Hemophilia A biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S. Food and Drug Administration (FDA) accepted the Company's resubmission of the Biologics License Application (BLA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. |
| 2022-09-29 | BioMarin Pharmaceutical | Regulatory BioMarin Resubmits Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Severe Hemophilia A to the FDA biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Company resubmitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. |
| 2022-08-24 | BioMarin Pharmaceutical | Regulatory First Gene Therapy for Adults with Severe Hemophilia A, BioMarin’s ROCTAVIAN™ (valoctocogene roxaparvovec), Approved by European Commission (EC) biomarin.com ↗ |
| 2022-07-11 | BioMarin Pharmaceutical | Results BioMarin Presents Findings from Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the International Society on Thrombosis and Haemostasis (ISTH) 2022 Congress July 9-12, Including 4 Oral and 2 Poster Presentations biomarin.com ↗ |
| 2022-06-24 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. |
| 2022-05-31 | BioMarin Pharmaceutical | Results BioMarin Announces Durable Hemostatic Efficacy Maintained Over 6 years in Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec, Investigational Gene Therapy for Hemophilia A biomarin.com ↗
Six-year and five-year post-treatment follow-up of the 6e13 vg/kg and 4e13 vg/kg cohorts, respectively, demonstrated sustained hemostatic efficacy of valoctocogene roxaparvovec. |
| 2022-04-27 | BioMarin Pharmaceutical | Results BioMarin Announces Record Revenues in First Quarter 2022 biomarin.com ↗
Today, BioMarin announced that the findings from the completed analysis showed a comparable pattern of integration between healthy and tumor containing tissues, with no evidence emerging that vector integration contributed to the salivary gland mass. |
| 2022-03-17 | BioMarin Pharmaceutical | Results BioMarin Announces Publication in New England Journal of Medicine of One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A biomarin.com ↗ |
| 2022-02-04 | BioMarin Pharmaceutical | Results BioMarin Announces Oral Presentation of 2-Year Analysis of Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A at 15th Annual Congress of European Association for Haemophilia and Allied Disorders (EAHAD) 2-4 February biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Company presented positive results from a two-year analysis of the Phase 3 GENEr8-1 study and an overall safety update of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the 15 th Annual Virtual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD). |
| 2022-01-09 | BioMarin Pharmaceutical | Results BioMarin Announces Stable and Durable Annualized Bleed Control in the Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A; 134-Participant Study Met All Primary and Secondary Efficacy Endpoints at Two Year Analysis biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. |
| 2021-07-21 | BioMarin Pharmaceutical | Results BioMarin Announces Oral Presentation at International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress with 5 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec in Adults with Severe Hemophilia A, Demonstrating Continued, Durable Clinical Benefit biomarin.com ↗ |
| 2021-07-19 | BioMarin Pharmaceutical | Results BioMarin Announces Oral Presentation of Positive One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A at International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress biomarin.com ↗ |
| 2021-07-15 | BioMarin Pharmaceutical | Regulatory European Medicines Agency Validates BioMarin’s Marketing Authorization Application for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. |
| 2021-07-02 | BioMarin Pharmaceutical | Results BioMarin Announces 12 Presentations at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress biomarin.com ↗
We are proud of the consistent and dramatic bleed control results to date, based on both long-term extension studies of at least five years, and the largest and most definitive gene therapy study in Hemophilia A. |
| 2021-06-28 | BioMarin Pharmaceutical | Regulatory BioMarin Resubmits Marketing Authorization Application (MAA) to European Medicines Agency for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. |
| 2021-05-24 | BioMarin Pharmaceutical | Regulatory European Medicines Agency Grants BioMarin’s Request for Accelerated Assessment of Valoctocogene Roxaparvovec for Treatment of Severe Hemophilia A biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) has granted its request for accelerated assessment of valoctocogene roxaparvovec, for adults with severe hemophilia A. |
| 2021-05-19 | BioMarin Pharmaceutical | Results BioMarin Provides Highlights of 5 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec with the Longest Duration of Clinical Experience for a Gene Therapy in Hemophilia A biomarin.com ↗
Five-year and four-year post-treatment follow-up of the 6e13 vg/kg and 4e13 vg/kg cohorts, respectively, shows a sustained treatment benefit of valoctocogene roxaparvovec. |
| 2021-03-08 | BioMarin Pharmaceutical | Regulatory BioMarin Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Valoctocogene Roxaparvovec, Investigational Gene Therapy for Hemophilia A biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. |
| 2021-01-10 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. |
| 2020-08-19 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Complete Response Letter (CRL) from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A biomarin.com ↗ |
| 2020-06-17 | BioMarin Pharmaceutical | Results BioMarin Provides Additional Data from Recent 4 Year Update of Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in Late-Breaking Oral Presentation at World Federation of Hemophilia Virtual Summit biomarin.com ↗
With four years of data, this study represents the longest duration of clinical experience for any gene therapy in hemophilia A. |
| 2020-05-31 | BioMarin Pharmaceutical | Results BioMarin Provides Highlights of 4 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A biomarin.com ↗
The four-year update for the 6e13 vg/kg and three-year update for the 4e13 vg/kg cohorts demonstrated that all subjects in both cohorts remain off prophylactic Factor VIII treatment since receiving their single dose of valoctocogene roxaparvovec. |
| 2020-02-20 | BioMarin Pharmaceutical | Regulatory BioMarin’s Biologics License Application for Valoctocogene Roxaparvovec Accepted for Priority Review by FDA with Review Action Date of August 21, 2020 biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) to the FDA for its investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A. |
| 2020-01-02 | BioMarin Pharmaceutical | Results BioMarin Announces New England Journal of Medicine Publishes 3 Years of Follow-up Data in Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A biomarin.com ↗
The NEJM article, "Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A", demonstrated that a single infusion of valoctocogene roxaparvovec "resulted in sustained, clinically relevant benefit, as measured by a substantial reduction in annualized rates of bleeding events and complete cessation of prophylactic factor VIII use in all 13 participants who had received 4e13vg/kg or 6e13 vg/kg." |
| 2019-12-23 | BioMarin Pharmaceutical | Regulatory European Medicines Agency Validates BioMarin’s Marketing Authorization Application for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. |
| 2019-12-23 | BioMarin Pharmaceutical | Regulatory BioMarin Submits Biologics License Application to U.S. Food and Drug Administration for Valoctocogene Roxaparvovec to Treat Hemophilia A biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A. |
| 2019-11-21 | BioMarin Pharmaceutical | Regulatory BioMarin Submits Marketing Authorization Application to European Medicines Agency for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. |
| 2019-11-14 | BioMarin Pharmaceutical | Results Poised for Significant Growth and Profitability, BioMarin Shares Company Highlights During R&D Day on November 14th in New York biomarin.com ↗
Valoctocogene roxaparvovec shows a transformational reduction in bleeding and need for Factor VIII infusions. |
| 2019-05-28 | BioMarin Pharmaceutical | Results BioMarin Provides 3 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A biomarin.com ↗
The three-year update demonstrated that bleed rate control with valoctocogene roxaparvovec 6e13 vg/kg dose was maintained for a third year with a median Annualized Bleed Rate (ABR) of 0 and mean ABR of 0.7 in that year. |
| 2019-05-28 | BioMarin Pharmaceutical | Results BioMarin Announces that Phase 3 Cohort of Valoctocogene Roxaparvovec, Gene Therapy Study in Severe Hemophilia A Met Pre-Specified Criteria for Regulatory Submissions in the U.S. and Europe biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A achieved pre-specified clinical criteria for regulatory review in the U.S. and Europe . |
| 2018-05-22 | BioMarin Pharmaceutical | Results BioMarin Provides 2 Years of Clinical Data in 6e13 vg/kg Dose from Ongoing Phase 1/2 Study in Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at World Federation of Hemophilia 2018 World Congress biomarin.com ↗ |
| 2017-12-11 | BioMarin Pharmaceutical | Results BioMarin Highlights New Results for Gene Therapy Valoctocogene Roxaparvovec at the 2017 American Society of Hemophilia (ASH) Meeting biomarin.com ↗
With the 6e13 vg/kg dose, at 78 weeks post infusion, the median and mean Factor VIII levels of the 6e13 vg/kg cohort were 90 and 89%, respectively. |
| 2017-12-09 | BioMarin Pharmaceutical | Results BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at 59th American Society of Hematology (ASH) Annual Meeting Concurrent with NEJM Publication biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. |
| 2017-10-26 | BioMarin Pharmaceutical | Regulatory FDA Grants Breakthrough Therapy Designation for BioMarin’s Valoctocogene Roxaparvovec (formerly BMN 270), an Investigational Gene Therapy for Hemophilia A biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S. Food and Drug Administration (FDA) granted valoctocogene roxaparvovec (formerly BMN 270) Breakthrough Therapy Designation. |
| 2017-10-18 | BioMarin Pharmaceutical | Results BioMarin Highlights Breadth of Innovative Development Pipeline at R&D Day on October 18th in New York biomarin.com ↗
BMN 270 Reduces Bleeds and Factor VIII Use |
All press releases naming this drug 53 releases
Evidence & citations 10 cited values
Every value below carries the sentence it was read from. 12 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Valoctocogene Roxaparvovec | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03392974 ↗ |
| Known as | AAV5-FVIII-SQ | “such as human FVIII-SQ in valoctocogene roxaparvovec (AAV5-FVIII-SQ).” PMID 32915950 ↗ Nov 2020 |
| Known as | AAV5-hFVIII-SQ | “Factor VIII gene transfer with a single intravenous infusion of valoctocogene roxaparvovec (AAV5-hFVIII-SQ) has demonstrated clinical benefits lasting 5 years to date in people...” PMID 35411075 ↗ Apr 2022 |
| Known as | BMN-270 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297579 ↗ |
| Known as | Roctavian | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297579 ↗ |
| Known as | Valoctocogen roxaparvovec | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297579 ↗ |
| Action | Restore | “Valoctocogene roxaparvovec is a single administration gene therapy treatment that enables endogenous factor VIII (FVIII) production to prevent bleeding in people with severe...” PMID 40986187 ↗ Sep 2025 |
| Modality | Gene therapy (AAV / viral vector) | “Valoctocogene roxaparvovec is an adeno-associated virus vector serotype 5 (AAV5)-mediated gene therapy approved for severe haemophilia A (HA).” PMID 38975624 ↗ Jul 2024 |
| Route | Intravenous | “Factor VIII gene transfer with a single intravenous infusion of valoctocogene roxaparvovec” PMID 35411075 ↗ Apr 2022 |
| Target | F9 | “Coagulation factor IX exogenous protein” CHEMBL4297579 ↗ |