Valoctocogene roxaparvovec

Overview

Valoctocogene roxaparvovec is an adeno-associated virus serotype 5 (AAV5) based gene therapy vector that expresses the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ). The expression of hFVIII-SQ is driven by a liver-specific promoter, which enables hepatocytes to produce factor VIII protein and increase the levels of active factor VIII in blood. Valoctocogene roxaparvovec was approved by EMA in September 2022 and is indicated for the treatment of severe hemophilia A. It is not approved for use in the United States. Hemophilia A treatments such as prophylactic regimens of exogenous factor VIII or emicizumab improve the clinical outcomes of patients but do not eliminate breakthrough bleeding. As opposed to these therapies, valoctocogene roxaparvovec offers the advantage of continuous and measurable steady-state levels of coagulation factor VIII.

Indication

Valoctocogene roxaparvovec is indicated for the treatment of severe hemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).

Associated Conditions

Severe Hemophilia A

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors	2020/12/28	NCT04684940	Phase 1	Active, not recruiting	BioMarin Pharmaceutical
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5	2018/05/11	NCT03520712	Phase 1	Active, not recruiting	BioMarin Pharmaceutical
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)	2017/12/13	NCT03370913	Phase 3	Completed	BioMarin Pharmaceutical
Gene Therapy Study in Severe Haemophilia A Patients (270-201)	2015/10/15	NCT02576795	Phase 1	Completed	BioMarin Pharmaceutical

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
ROCTAVIAN	BioMarin Pharmaceutical Inc.	68135-927	INTRAVENOUS	20000000000000 {GC} in 1 mL	3/4/2024

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Roctavian	BioMarin International Limited,Shanbally,Ringaskiddy,County Cork,P43 R298,Ireland	Authorised	8/24/2022

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
No TGA approvals found for this drug.

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
No Health Canada approvals found for this drug.

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
No CIMA AEMPS (Spain) approvals found for this drug.

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

Related News

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