---
title: "BioMarin Pharmaceutical Reports Sustained Efficacy for Hemophilia A Gene Therapy in Five-Year Data"
type: "News"
locale: "en"
url: "https://longbridge.com/en/news/245927715.md"
description: "BioMarin Pharmaceutical said Tuesday that new five-year data from its Phase 3 study showed roctavian maintained bleed control and factor VIII expression in adults with severe hemophilia A. The trial found 81.3% of participants remained off preventive treatment and 77.8% had no treated bleeds in the fifth year, the company said. No new safety signals were observed, and no participants developed inhibitors, it added."
datetime: "2025-06-24T13:55:17.000Z"
locales:
  - [zh-CN](https://longbridge.com/zh-CN/news/245927715.md)
  - [en](https://longbridge.com/en/news/245927715.md)
  - [zh-HK](https://longbridge.com/zh-HK/news/245927715.md)
---

> Supported Languages: [简体中文](https://longbridge.com/zh-CN/news/245927715.md) | [繁體中文](https://longbridge.com/zh-HK/news/245927715.md)


# BioMarin Pharmaceutical Reports Sustained Efficacy for Hemophilia A Gene Therapy in Five-Year Data

BioMarin Pharmaceutical said Tuesday that new five-year data from its Phase 3 study showed roctavian maintained bleed control and factor VIII expression in adults with severe hemophilia A.

The trial found 81.3% of participants remained off preventive treatment and 77.8% had no treated bleeds in the fifth year, the company said.

No new safety signals were observed, and no participants developed inhibitors, it added.

### Related Stocks

- [BioMarin Pharmaceutical Inc. (BMRN.US)](https://longbridge.com/en/quote/BMRN.US.md)

## Related News & Research

- [Glenmark goes direct with new US Ryaltris marketing plan](https://longbridge.com/en/news/281533476.md)
- [Glenmark Pharma to directly commercialize and distribute Ryaltris in U.S. effective](https://longbridge.com/en/news/281370208.md)
- [BioMarin: Sustained Double-Digit Growth Story Underpinned by Voxzogo Leadership, Advancing Rare-Disease Pipeline, and Accretive Amicus Acquisition](https://longbridge.com/en/news/272411009.md)
- [Balanced View on BioMarin’s Amicus Acquisition: Strategic Rare-Disease Upside Versus Near-Term Leverage and Earnings Dilution Supports Hold Rating](https://longbridge.com/en/news/270339367.md)
- [GEN secures BEBO Foundation approval for Phase II PD trial](https://longbridge.com/en/news/279413854.md)