Biomarin’s hemophilia gene therapy nears European market after US setback

A single dose of the US-based biotech firm’s drug could be enough for a lifelong treatment of the rare bleeding disorder – but Roctavian’s price tag is making governments around the world think twice.
Photo: Peter Dejong/AP/Ritzau Scanpix
Photo: Peter Dejong/AP/Ritzau Scanpix
By James Paton, bloomberg

Biomarin Pharmaceutical Inc. expects European patients to begin receiving its gene therapy for a life-threatening blood-clotting disorder before the end of the year, raising the drugmaker’s hopes it can succeed where rival Bluebird Bio Inc. faltered.

Already a subscriber?Log in here

Read the whole article

Get access for 14 days for free. No credit card is needed, and you will not be automatically signed up for a paid subscription after the free trial.

With your free trial you get:

  • Access all locked articles
  • Receive our daily newsletters
  • Access our app
!
!
Must contain at least 6 characters
!
Must contain at least 2 characters
!
Must contain at least 2 characters

Get full access for you and your coworkers

Start a free company trial today

Sign up for our newsletter

Stay ahead of development by receiving our newsletter on the latest sector knowledge.

!
Newsletter terms

Front page now

Further reading