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

Drug Profile

Valoctocogene roxaparvovec - BioMarin Pharmaceutical

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

Latest Information Update: 14 Jun 2024

Price : $50 *
  • 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

  • Marketed Haemophilia A

Most Recent Events

  • 07 Jun 2024 Efficacy and adverse events data from a phase III GENEr8-1 trial in Haemophilia A released by BioMarin Pharmaceutical
  • 07 Jun 2024 Efficacy data from a phase I/II trial in Haemophilia A released by BioMarin Pharmaceutical
  • 24 Apr 2024 Launched for Haemophilia A in Italy (IV)
Subscriber content

You need to be a logged in subscriber to view this content.

If your organization has a subscription then there are several options available to help you access AdisInsight, even while working remotely.

  • IP authentication when working within your organization’s network.
  • with username/password or try via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days.
  • Contact us at AsktheExpert.AdisInsight@springer.com for assistance.

If your organization does not have a subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Buy a PDF version of the profile.
  • Request a free trial
Back to top