BMN 270

Drug Profile

BMN 270

Alternative Names: AAV5-FVIII Gene Transfer - BioMarin; BMN-270

Latest Information Update: 20 May 2017

Price : $50

At a glance

  • Originator St. Jude Childrens Research Hospital; University College London
  • Developer BioMarin Pharmaceutical
  • Class Blood coagulation factors; Gene therapies
  • Mechanism of Action Blood coagulation factor replacements; Factor X stimulants; 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

  • Phase I/II Haemophilia A

Most Recent Events

  • 11 Jul 2017 BioMarin plans a phase III trial for Haemophilia A in USA
  • 08 Jan 2017 Efficacy and adverse event data from a phase I/II trial in Haemophilia A released by BioMarin Pharmaceutical
  • 13 Oct 2016 The Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom approves resumption of enrolment in a phase I/II trial for Haemophilia A
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at so we can help.

Back to top