💎 Fed’s first rate cut since 2020 set to trigger market. Find undervalued gems with Fair ValueSee Undervalued Stocks

Late-stage study underway for BioMarin's gene therapy for hemophilia A

Published 12/19/2017, 04:42 PM
© Reuters.  Late-stage study underway for BioMarin's gene therapy for hemophilia A
BMRN
-
  • The first patient has been dosed in a Phase 3 clinical trial, GENEr8-1, assessing BioMarin Pharmaceutical's (NASDAQ:BMRN) gene therapy valoctocogene roxaparvovec (VR) in patients with hemophilia A.
  • The study is the first of two registration trials that will support U.S. and European marketing applications. The second study, GENEr8-2, will assess a lower dose of VR. It should launch next quarter. According to ClinicalTrials.gov, the estimated primary completion date for GENEr8-1 is December 2022.
  • VR has Breakthrough Therapy and Orphan Drug status in the U.S. and Orphan Drug and PRIME status in Europe.
  • Now read: Biotech Analysis Central Pharma News: Bluebird Bio's Success, Spark's Hemophilia Data, Verastems Results


Original article

Latest comments

Risk Disclosure: Trading in financial instruments and/or cryptocurrencies involves high risks including the risk of losing some, or all, of your investment amount, and may not be suitable for all investors. Prices of cryptocurrencies are extremely volatile and may be affected by external factors such as financial, regulatory or political events. Trading on margin increases the financial risks.
Before deciding to trade in financial instrument or cryptocurrencies you should be fully informed of the risks and costs associated with trading the financial markets, carefully consider your investment objectives, level of experience, and risk appetite, and seek professional advice where needed.
Fusion Media would like to remind you that the data contained in this website is not necessarily real-time nor accurate. The data and prices on the website are not necessarily provided by any market or exchange, but may be provided by market makers, and so prices may not be accurate and may differ from the actual price at any given market, meaning prices are indicative and not appropriate for trading purposes. Fusion Media and any provider of the data contained in this website will not accept liability for any loss or damage as a result of your trading, or your reliance on the information contained within this website.
It is prohibited to use, store, reproduce, display, modify, transmit or distribute the data contained in this website without the explicit prior written permission of Fusion Media and/or the data provider. All intellectual property rights are reserved by the providers and/or the exchange providing the data contained in this website.
Fusion Media may be compensated by the advertisers that appear on the website, based on your interaction with the advertisements or advertisers.
© 2007-2024 - Fusion Media Limited. All Rights Reserved.