Global Blood Therapeutics, Inc.GBT announced that the FDA has agreed to its proposal of an accelerated approval pathway for its sickle cell disease ("SCD") candidate, voxelotor.
The company will file a new drug application ("NDA") under this pathway. The company anticipates that a rise in hemoglobin levels on treatment with voxelotor is likely to lead to reduction in strokes in SCD patients.
Per the discussion with the FDA, the company stated that the regulatory body has agreed to a post-approval confirmatory study to demonstrate stroke risk reduction with transcranial doppler flow velocity as its primary endpoint.
Accelerated approval is generally granted to therapies for severe and life-threatening conditions that address significant unmet medical needs. It is a conditional approval that will help the company to bring the drug faster in the market.
The company has made plans to request a pre-NDA meeting with the FDA in the first quarter of 2019. The company will announce the timing for submission of the NDA and specifics of the confirmatory study following the meeting.
Shares of Global Blood Therapeutics rallied 47.8% on Dec 3 following the news. The company's shares have gained 18.5% so far this year against the industry 's decline of 15.3%.
The company is developing voxelotor as a once-daily, oral therapy for improving anemia in patients with SCD, thereby preserving brain function and reducing strokes.
A phase III study - HOPE - and a phase IIa study - HOPE-KIDS 1 - are evaluating voxelotor in SCD patients aged 12 years and older and patients between 4 and 17 years of age, respectively.
Preliminary data from the late-stage study presented at the annual meeting of the American Society of Hematology (ASH) earlier this week demonstrated that the candidate achieved rapid, robust and sustained improvements in hemoglobin levels over 24 weeks of treatment. The increase in hemoglobin levels was clinically meaningful and statistically significant. These data were the key factors that led the FDA to accept the proposal.
We note that treatment options are limited for SCD. This increases the likelihood of success for voxelotor, upon potential approval. Last year, the FDA approved privately-held Emmaus Medical's Endari as a new treatment option for SCD in almost two decades. Meanwhile, Novartis NVS and bluebird bio, Inc. BLUE are developing crizanlizumab and LentiGlobin, respectively, for treating SCD in early to mid-stage clinical studies.
Global Blood Therapeutics, Inc. Price
Global Blood Therapeutics, Inc. Price | Global Blood Therapeutics, Inc. Quote
Zacks Rank and Stock to Consider
Global Blood Therapeutics currently carries a Zacks Rank #3 (Hold). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here .
A better-ranked from the biotech stock is Alexion Pharmaceuticals, Inc. ALXN with a Zacks Rank #1. The company's earnings estimates have increased from $7.23 to $7.61 for 2018 and from $8.59 to $8.77 for 2019 over the past 60 days. The company delivered positive earnings surprise in all the trailing four quarters with the average beat being 16.77%.
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