Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

Valoctocogene roxaparvovec - BioMarin Pharmaceutical

Next Previous
Drug Profile

Valoctocogene roxaparvovec - BioMarin Pharmaceutical

Alternative Names: AAV5-hFVIII-SQ; BMN-270; Factor-VIII-gene-therapy-BioMarin; Roctavian; V-ROX

Latest Information Update: 24 Jul 2026

Price :
$50 *
Note:
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

At a glance

  • Originator St. Jude Childrens Research Hospital; University College London
  • Developer BioMarin Pharmaceutical
  • Class Antihaemorrhagics; Blood coagulation factors; Gene therapies
  • Mechanism of Action Factor VIII replacements; Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Haemophilia A
  • New Molecular Entity No

Highest Development Phases

  • Registered Haemophilia A

Most Recent Events

  • 20 Apr 2026 BioMarin Pharmaceutical completes a phase I/II trials in Haemophilia A in US, Brazil, Italy, Turkey, South Korea and Taiwan(IV) (NCT04684940)
  • 24 Feb 2026 Withdrawn for Haemophilia A in Germany, Italy (IV)
  • 24 Jun 2025 Adverse events and efficacy data from the phase-III GENEr8-1 trial in Haemophilia A released by BioMarin Pharmaceutical

You need to be a logged in or subscribed to view this content

Request demo (opens in a new window)
If your organization or you do not have a subscription, try one of the following: If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top