Global Blood Therapeutics, Inc. (GBT - Free Report) 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 industry’s decline of 4.3%.
We note that treatment options are limited for SCD. However, Global Blood is likely to face stiff competition as many companies are developing therapies to treat the disease. In July 2019, the FDA accepted Novartis’ (NVS - Free Report) biologics license application (BLA) for its investigational medicine crizanlizumab (SEG101) to prevent pain crises (also called vaso-occlusive crises or VOCs) in patients with SCD and granted a priority review to the same. bluebird bio (BLUE - Free Report) is also developing a LentiGlobin BB305 gene therapy for the treatment of VOC in patients with SCD.
Zacks Rank and Key Pick
Global Blood currently has a Zacks Rank #3 (Hold). A better-ranked stock in the healthcare sector is Trevena, Inc. (TRVN - Free Report) , sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Trevena’s loss per share estimates have been narrowed 27% for 2019 and 19.5% for 2020 over the past 60 days. The stock has skyrocketed 94.3% year to date.
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