FDA Accepts NDA for Global Blood Therapeutics' Sickle

Cell Drug; Shares Up 14%

Streetwise Reports 09/05/2019

Streetwise Reports LLC

Shares of Global Blood Therapeutics are trading more than 14% higher after
101 Second St., Suite 110
Petaluma, CA 94952 the FDA accepted its New Drug Application filing for Voxelotor, a drug used
Tel.: (707) 981-8999 in the treatment of sickle cell disease.
Fax: (707) 773-5020
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Source: Streetwise Reports

STREETWISE REPORTS Earlier today clinical-stage biopharmaceutical firm Global Blood Therapeutics Inc.
THE ENERGY REPORT
(GBT:NASDAQ) announced that the U.S. Food and Drug Administration (FDA) has
accepted for filing the company's New Drug Application (NDA) seeking accelerated
THE GOLD REPORT
approval for voxelotor, an oral, once-daily therapy in development for the treatment
THE LIFE SCIENCES REPORT
of sickle cell disease (SCD). If approved, voxelotor would be the first therapy
available to patients that targets hemoglobin polymerization, the root cause of SCD
damage.

This timely announcement comes as September is designated as National Sickle

Cell Awareness month. The company states that it is proud to show its support for
the approximately 100,000 Americans affected by SCD. Together with the Sickle
Cell Disease Association of America Inc., Global Blood Therapeutics will be hosting
the 8th Annual Sickle Cell Disease Therapeutics Conference on September 10,
2019, in Washington, D.C.

In today's release, the firm noted that the FDA has granted Priority Review for the
NDA for voxelotor, which provides for a six-month review, and assigned a
Prescription Drug User Fee Act (PDUFA) target action date of February 26, 2020.
The agency also indicated in the NDA filing acceptance notification letter that it is
not currently planning to hold an advisory committee meeting to discuss the
application for voxelotor.

Ted W. Love, M.D., the company's president and CEO, stated, "The FDA's
acceptance of our NDA for voxelotor under Priority Review is a major milestone in
the development of this investigational therapy and further illustrates the
significance the Agency places on getting important and innovative treatments to
individuals living with SCD as quickly as possible...We look forward to working with
the FDA during this process, with the goal of potentially changing the treatment
paradigm for SCD."

The company advised that Priority Review is granted to therapies that the FDA
determines have the potential to provide a significant improvement in the safety or
effectiveness of the treatment, diagnosis or prevention of a serious condition.
Under the PDUFA, a Priority Review targets an accelerated review time of six
months compared to a standard review time of 10 months.

The firm states that the NDA for voxelotor is supported by data from the multi-
national Phase 3 HOPE Study of voxelotor in patients ages 12 and older with SCD
who enrolled in the study from 60 institutions across 12 countries. The company
indicated that the HOPE Study results were recently published in The New England
Journal of Medicine, and that voxelotor has been granted FDA Breakthrough
Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the
treatment of patients with SCD.

The company explains that Sickle Cell Disease (SCD) is a lifelong inherited blood
disorder caused by a genetic mutation in the beta-chain of hemoglobin, which
leads to the formation of abnormal hemoglobin known as sickle hemoglobin (HbS).
Beginning in childhood, SCD patients typically suffer unpredictable and recurrent
 episodes or crises of severe pain due to blocked blood flow to organs, which often
lead to psychosocial and physical disabilities.

Global Blood Therapeutics is based in South San Francisco, Calif., and describes
its business as a clinical-stage biopharmaceutical company determined to discover,
develop and deliver innovative treatments that provide hope to underserved patient
communities. The firm is also developing another therapy for the potential
treatment of sickle cell disease.

GBT shares opened higher today at $54.45 (+$6.96, +14.66%) over yesterday's
close of $47.49. The stock has traded today between $52.61 and $56.50/share and
is currently trading at $54.42 (+$6.93, +14.59%).

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