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NCT06224907Phase 3ACTIVE NOT RECRUITING

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

Study Summary

This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.

Conditions
Hemophilia A
Interventions / Drugs
Valoctocogene roxaparvovec
View on ClinicalTrials.gov ↗
Sponsor
BMRN
BioMarin Pharmaceutical
Rare Disease
D49/100
Study Details
Enrollment6 participants
Primary Completion—
Lead SponsorBioMarin Pharmaceutical
Data Retrieved2026-04-11 02:55:23.531364+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 6 participants
Sponsor Catalyst Profile

BioMarin Pharmaceutical (BMRN) is the sponsoring company for this trial. BiotechSign currently grades this company D (49/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

BMRN Full Pipeline →All Companies →
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