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Roctavian (VALOCTOCOGENE ROXAPARVOVEC)
Roctavian (generic name: VALOCTOCOGENE ROXAPARVOVEC) is a Gene therapy drug developed by BioMarin International Limited. It is currently FDA-approved (first approved 2022) for Severe hereditary factor VIII deficiency disease.
Roctavian introduces a functional copy of the F8 gene into cells to produce factor VIII protein.
Roctavian (valoctocogene roxaparvovec) is a gene therapy developed by BioMarin International Limited for the treatment of severe hereditary factor VIII deficiency disease. It works by introducing a functional copy of the F8 gene into the patient's cells, allowing them to produce factor VIII protein. Roctavian is currently patented and has been approved by the FDA for its intended use. Key safety considerations include the risk of insertional mutagenesis and the potential for immune responses against the vector. In plain terms, Roctavian helps patients with severe factor VIII deficiency by giving their cells the instructions to make the missing protein.
At a glance
| Generic name | VALOCTOCOGENE ROXAPARVOVEC |
|---|---|
| Sponsor | BioMarin International Limited |
| Modality | Gene therapy |
| Therapeutic area | Rare Disease |
| Phase | FDA-approved |
| First approval | 2022 |
Mechanism of action
Valoctocogene roxaparvovec-rvox is an adeno-associated virus serotype (AAV5) based gene therapy vector, designed to introduce functional copy of transgene encoding the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ). Transcription of this transgene occurs within the liver, using liver-specific promoter, which results in the expression of hFVIII-SQ. The expressed hFVIII-SQ replaces the missing coagulation factor VIII needed for effective hemostasis.
Approved indications
- Severe hereditary factor VIII deficiency disease
Common side effects
- ALT increases ULN
- AST increases ULN
- LDH increases ULN
- CPK increases ULN
- Factor VIII activity levels ULN
- GGT increases ULN
- Bilirubin increases ULN
- Nausea
- Fatigue
- Infusion-related reactions
- Headache
- Vomiting
Key clinical trials
- Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (PHASE3)
- Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (PHASE3)
- Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 (PHASE1,PHASE2)
- Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors (PHASE1,PHASE2)
- Gene Therapy Study in Severe Haemophilia A Patients (270-201) (PHASE1,PHASE2)
- Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) (PHASE3)
- Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg (PHASE3)
Patents
| Patent | Expiry | Type |
|---|---|---|
| Biologic Exclusivity |
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Roctavian CI brief — competitive landscape report
- Roctavian updates RSS · CI watch RSS
- BioMarin International Limited portfolio CI
Frequently asked questions about Roctavian
What is Roctavian?
How does Roctavian work?
What is Roctavian used for?
Who makes Roctavian?
What is the generic name of Roctavian?
When was Roctavian approved?
What development phase is Roctavian in?
What are the side effects of Roctavian?
Related
- Manufacturer: BioMarin International Limited — full pipeline
- Therapeutic area: All drugs in Rare Disease
- Indication: Drugs for Severe hereditary factor VIII deficiency disease
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing