Skip to main content

Market Overview

BioMarin's Hemophilia Gene Therapy Scores RMAT Designation In US

Share:

The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BioMarin Pharmaceutical Inc's (NASDAQ: BMRN) valoctocogene roxaparvovec investigational gene therapy for the treatment of adults with severe hemophilia A. 

  • RMAT is an expedited program intended to facilitate the development and review of regenerative medicine therapies.
  • In January, Phase 3 data were reported from patients with five bleeding episodes per year and using about 136-factor infusions per year.
  • Gene therapy treatment cut those average annualized numbers by 84% and 99%, respectively, to just under one and two.
  • In August last year, the Company received FDA Complete Response Letter for valoctocogene roxaparvovec.
  • The agency recommended that the Company complete the Phase 3 GENEr8-1 study and submit two-year follow-up safety and efficacy data.
  • In the EU, BioMarin is targeting submission of the marketing application with these results to the EMA in the second quarter of 2021, pending confirmation in planned pre-submission meetings.
  • Price Action: BMRN shares gained 0.66% at $78.22 in market trading hours on the last check Monday.
 

Related Articles (BMRN)

View Comments and Join the Discussion!

Posted-In: gene therapy Hemophilia ABiotech News Health Care FDA General

Don't Miss Any Updates!
News Directly in Your Inbox
Subscribe to:
Benzinga Premarket Activity
Get pre-market outlook, mid-day update and after-market roundup emails in your inbox.
Market in 5 Minutes
Everything you need to know about the market - quick & easy.
Fintech Focus
A daily collection of all things fintech, interesting developments and market updates.
SPAC
Everything you need to know about the latest SPAC news.
Thank You

Thank you for subscribing! If you have any questions feel free to call us at 1-877-440-ZING or email us at vipaccounts@benzinga.com