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Global Blood's Sickle Cell Disease Drug Snags Accelerated FDA Approval Pathway

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Global Blood's Sickle Cell Disease Drug Snags Accelerated FDA Approval Pathway
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Global Blood Therapeutics Inc (NASDAQ: GBT) shares are trading at a nearly three-month high, with a FDA event being the catalyst.

What Happened

Global Blood announced Monday that the FDA has agreed to allow filing for its late-stage sickle cell disease, or SCD, candidate Voxelotor under an accelerated approval pathway.

The company said it would submit a new drug application, or NDA, for the pipeline asset under the pathway.

With Global Blood proposing that voxelotor reasonably reduces strokes in SCD patients, the FDA has accepted the transcranial doppler, or TCD, flow as the primary endpoint in a post-approval confirmatory study to demonstrate stroke risk reduction.

"These events reiterate our belief that voxelotor's improvement to hemoglobin reflects a clinically meaningful benefit for patients, has a clear path to accelerated approval, and is well-positioned with its Phase 3 HOPE study," Cantor Fitzgerald analyst Elemer Piros said in a note. 

"Based on the existing clinical data, we believe voxelotor offers a compelling treatment option in SCD that lacks disease-modifying therapies."

Why It's Important

The accelerated approval under Subpart H is allowed for drugs that treat serious or life-threatening illnesses and provide meaningful therapeutic benefit to patients over existing treatments.

The superiority has to be established through clinical trials that confirm the drug product has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit.

"A once-daily oral therapy that has the potential to safely improve the anemia of SCD and thereby preserve brain function would be a major breakthrough. I am encouraged by the FDA's acknowledgement of the urgent need for new treatment options for the SCD community," Kim Smith-Whitley, director of the Comprehensive Sickle Cell Center at the Children's Hospital of Philadelphia, said in a statement. 

What's Next

Global Blood said it plans to request a pre-NDA meeting in the first quarter of 2019. The company said it would provide further details on the plans and timing of NDA submission as well as additional specifics on the TCD confirmatory study following this meeting.

Cantor Fitzgerald has an Overweight rating and $96 price target for Global Blood.

Global Blood shares were rallying 44.74 percent to $45.65 at the time of publication Monday. 

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Latest Ratings for GBT

DateFirmActionFromTo
Aug 2018PiperJaffrayInitiates Coverage OnOverweight
Aug 2018WedbushMaintainsOutperformOutperform
Jun 2018Cantor FitzgeraldMaintainsOverweightOverweight

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