FDA Grants Priority Review To Global Blood's SCD Candidate
Zacks Investment Research | Sep 05, 2019 11:53PM ET
Global Blood Therapeutics, Inc. (NASDAQ:GBT) announced that the FDA has accepted its new drug application (NDA) for its lead candidate voxelotor, which has been developed for the treatment of sickle cell disease (SCD).
With the FDA granting a priority review to the NDA, a decision from the regulatory body is expected on Feb 26, 2020.
The NDA was based on data from the phase III HOPE study, which evaluated voxelotor as an oral, once-daily therapy for SCD in patients aged 12 years and above. In the study, treatment with voxelotor led to statistically significant and sustained improvements in hemoglobin levels.
SCD is a chronic, inherited blood disorder that affects hemoglobin levels. Upon potential approval, voxelotor is poised to become the first treatment available to patients that targets hemoglobin polymerization, a major cause for SCD damage.
The priority review for voxelotor will reduce its review time to six months compared with the standard review time of 10 months. Notably, last December, Global Blood announced that the FDA agreed to its proposal for an accelerated approval pathway for voxelotor.
Voxelotor has also been previosuly granted a Fast Track, Orphan Drug and Breakthrough Therapy designations by the FDA for the treatment of SCD.
Meanwhile, voxelotor is also being evaluated in the ongoing phase IIa HOPE-KIDS 1 study for treating SCD in pediatric patients aged four to 17 years. The single and multiple-dose study is assessing the safety, tolerability, pharmacokinetics and the exploratory treatment effect of voxelotor in children.
Shares of Global Blood have rallied 28.3% so far this year against the Zacks Investment Research
Get The News You Want
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.