Action Required: Password Reset on Next Login

As part of our recent security and system improvements, a password reset will be required the next time you log in. This applies to username/password users only; SSO users are not affected.

To reset your password, please visit the Reset Password page.

We appreciate your patience during this transition and are available to assist with any questions. For support, please call us at +1 650-552-4224 or email us at support@biocentury.com.

BioCentury
ARTICLE | Regulation

What FDA’s request for a new endpoint means for BioMarin’s hemophilia gene therapy

Collecting two-year data will set BioMarin back at least 15 months, but it still has a shot at getting its gene therapy to market first

August 20, 2020 1:50 AM UTC

FDA has moved the goal post for BioMarin’s Roctavian, but the candidate could still be the first gene therapy to win approval for hemophilia A.

Shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) tumbled $41.83 (35%) to $76.71 Wednesday, losing $7.6 billion in market value, after FDA issued a complete response letter for Roctavian valoctocogene roxaparvovec. The company plans to meet with FDA in the coming weeks to align on next steps for the adeno-associated virus serotype 5 (AAV5) vector delivering Factor VIII. The therapy, which was evaluated under Priority Review, has breakthrough therapy and Orphan Drug designation. ...

Get Unlimited Access
Continue reading with a free trial.
Or Purchase This Article

BCIQ Company Profiles

Biomarin Pharmaceutical Inc.

BCIQ Target Profiles

Factor VIII