Its Gene Therapy Roctavian Finally Approved With $2.9m Price, BioMarin Aims To Prove Doubters Wrong

FDA Label Muddies Efficacy Claims

BioMarin has finally brought its hemophilia A gene therapy Roctavian market and is determined prove naysayers wrong, but must work with its limitations and a confusing US label.

• Source: Shutterstock

BioMarin has gained US approval for Roctavian, the first ever hemophilia A gene therapy, and the company is confident it can prove doubters wrong about how popular the one-time treatment will be with patients and payers.

However, the high $2

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

Start your 7-day free trial
Explore trusted news, analysis, and insights
Access comprehensive global coverage
Enjoy instant access – no credit card required

Start Free Trial

Already a subscriber?

Most Favored Nation Policy ‘Lazy, Misguided,’ But US Price Reforms Politically ‘Inevitable’

15 May 2025

• By Cathy Kelly

Policy experts and pharma executives react to President Trump’s executive order on “equalizing” drug prices between the US and comparable nations at the ISPOR 2025 conference.