Additional data demands delay Biomarin's hemophilia drug submission in US

The US health authority has requested more data pertaining to the company’s hemophilia A treatment candidate, and Biomarin has subsequently postponed its marketing approval submission.

Photo: Manuel Balce Ceneta/AP/Ritzau Scanpix

Gene therapy valoctocogene roxaparvovec will not be up for re-evaluation by the US Food and Drug Administration (FDA) until September, biotech firm Biomarin has announced in a press release.

In the same statement, Biomarin also reports that no patient who received the highest drug dose has required the preventive hemophilia treatment known as prophylactic Factor VIII in the six years since they started the firm’s gene therapy.

Already a subscriber? Log in.

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.

  • Access all locked articles
  • Receive our daily newsletters
  • Access our app
An error has occured. Please try again later.

Get full access for you and your coworkers.

Start a free company trial today

More from MedWatch

Further reading

Related articles

Latest news

See all jobs