valoctocogene roxaparvovec

Pipeline

Status by Indication[3][1]

Competition

Competitive Landscape

7 competitors in Severe Hemophilia a

#DrugCompanyPhaseTrials

1

Hemlibra

Roche

Approved

42

FRSW107

KKaifeng Pharmaceutical (Group) Co., Ltd.

Phase 3

33

valoctocogene roxaparvovec

BioMarin Pharmaceutical

Phase 3

24

Marstacimab

Pfizer

Phase 1

1

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Clinical

Trials by Indication[1]

Hemophilia A3 trials

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

Completed·BioMarin Pharmaceutical·Phase 3·NCT04323098·22 enrolled

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

Completed·BioMarin Pharmaceutical·Phase 3·NCT03392974·1 enrolled

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

Completed·BioMarin Pharmaceutical·Phase 3·NCT03370913·144 enrolled

Severe Hemophilia a2 trials

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

Active, not recruiting·BioMarin Pharmaceutical·Phase 3·NCT06224907·6 enrolled

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

Terminated·BioMarin Pharmaceutical·Phase 1/2·NCT03520712·3 enrolled

Haemophilia A1 trial

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

Completed·BioMarin Pharmaceutical·Phase 1/2·NCT02576795·15 enrolled

Hemophilia A With Inhibitor1 trial

A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors

Completed·BioMarin Pharmaceutical·Preclinical·NCT04684940·10 enrolled

Details

Drug Info

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