GBT Receives FDA Breakthrough Therapy Designation for Voxelotor for Treatment of Sickle Cell Disease (SCD)
January 09 2018 - 08:00AM
Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced
that the U.S. Food and Drug Administration (FDA) has granted
Breakthrough Therapy Designation (BTD) to voxelotor (previously
called GBT440) for the treatment of sickle cell disease (SCD).
Voxelotor is being developed as a disease-modifying therapy for SCD
and previously received European Medicines Agency (EMA) Priority
Medicines (PRIME) designation for the treatment of SCD.
“The FDA’s decision to grant voxelotor the first Breakthrough
Therapy designation for the treatment of sickle cell disease
reflects a recognition of the promising efficacy and safety data we
have collected to date for this investigational drug, as well as an
acknowledgement of the overwhelming need for major advances over
available therapies in the treatment of SCD patients,” said Ted W.
Love, president and chief executive officer of GBT. “This
designation is another significant milestone for GBT as we work to
expedite the development of voxelotor.”
The FDA selectively grants BTD to expedite the development and
review of drugs that have demonstrated preliminary clinical
evidence indicating the potential for substantial improvement over
available therapy. The BTD decision for voxelotor was based on
clinical data submitted from the following studies:
- Preliminary efficacy and safety data from Part A of the Phase 3
HOPE Study (GBT440-031)
- Phase 1/2 study and open-label extension in adults
(GBT440-001/024)
- Ongoing Phase 2 HOPE-KIDS 1 study in children age 6 to 17
(GBT440-007)
- Compassionate Access experience in adults with severe SCD (not
eligible for the HOPE Study)
About Sickle Cell Disease (SCD)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). In its
deoxygenated state, HbS has a propensity to polymerize, or bind
together, forming long, rigid rods within a red blood cell (RBC).
The polymer rods deform RBCs to assume a sickled shape and to
become inflexible, which can cause blockage in capillaries and
small blood vessels. Beginning in childhood, SCD patients 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. This blocked blood flow, combined with
hemolytic anemia (the destruction of RBCs), can eventually lead to
multi-organ damage and early death.
About Voxelotor in Sickle Cell DiseaseVoxelotor
(previously called GBT440) is being developed as an oral,
once-daily therapy for patients with SCD. Voxelotor works by
increasing hemoglobin's affinity for oxygen. Since oxygenated
sickle hemoglobin does not polymerize, GBT believes voxelotor
blocks polymerization and the resultant sickling of red blood
cells. With the potential to restore normal hemoglobin function and
improve oxygen delivery, GBT believes that voxelotor may
potentially modify the course of SCD. In recognition of the
critical need for new SCD treatments, the U.S. Food and Drug
Administration (FDA) has granted voxelotor Fast Track, Orphan Drug
and Rare Pediatric Disease designations for the treatment of
patients with SCD. The European Medicines Agency (EMA) has included
voxelotor in its Priority Medicines (PRIME) program, and the
European Commission (EC) has designated voxelotor as an orphan
medicinal product for the treatment of patients with SCD.
GBT is currently evaluating voxelotor in the HOPE
(Hemoglobin Oxygen Affinity
Modulation to Inhibit HbS
PolymErization) Study, a Phase 3
clinical study in patients age 12 and older with SCD. Additionally,
voxelotor is being studied in the ongoing Phase 2a HOPE-KIDS 1
Study, an open-label, single- and multiple-dose study in pediatric
patients (age 6 to 17) with SCD. HOPE-KIDS 1 is assessing the
safety, tolerability, pharmacokinetics and exploratory treatment
effect of voxelotor.
About GBTGBT is a clinical-stage
biopharmaceutical company determined to discover, develop and
deliver innovative treatments that provide hope to underserved
patient communities. GBT is developing its lead product candidate,
voxelotor, as an oral, once-daily therapy for sickle cell disease.
To learn more, please visit www.gbt.com and follow the company on
Twitter @GBT_news.
Forward-Looking Statements Statements we make
in this press release may include statements that are not
historical facts and are considered forward-looking within the
meaning of Section 27A of the Securities Act of 1933, as amended
and Section 21E of the Securities Exchange Act of 1934, as amended.
We intend these forward-looking statements, including statements
regarding the therapeutic potential and safety profile of voxelotor
(previously called GBT440), our ability to implement and complete
our clinical development plans for voxelotor, our ability to
generate and report data from our ongoing and potential future
studies of voxelotor (including our ongoing Phase 3 HOPE Study and
our ongoing Phase 2a HOPE-KIDS 1 Study), regulatory review and
actions relating to voxelotor, and the timing of these events, to
be covered by the safe harbor provisions for forward-looking
statements contained in Section 27A of the Securities Act and
Section 21E of the Securities Exchange Act and are making this
statement for purposes of complying with those safe harbor
provisions. These forward-looking statements reflect our current
views about our plans, intentions, expectations, strategies and
prospects, which are based on the information currently available
to us and on assumptions we have made. We can give no assurance
that the plans, intentions, expectations or strategies will be
attained or achieved, and furthermore, actual results may differ
materially from those described in the forward-looking statements
and will be affected by a variety of risks and factors that are
beyond our control including, without limitation, the risks that
our clinical and preclinical development activities may be delayed
or terminated for a variety of reasons, that results of clinical
trials may be subject to differing interpretations, that regulatory
authorities may disagree with our clinical development plans or
require additional studies or data to support further clinical
investigation of our product candidates, that drug-related adverse
events may be observed in clinical development, and that data and
results may not meet regulatory requirements or otherwise be
sufficient for further development, regulatory review or approval,
along with those risks set forth in our Annual Report on Form 10-K
for the fiscal year ended December 31, 2016, and in our Quarterly
Report on Form 10-Q for the quarter ended September 30, 2017, as
well as discussions of potential risks, uncertainties and other
important factors in our subsequent filings with the U.S.
Securities and Exchange Commission. Except as required by law, we
assume no obligation to update publicly any forward-looking
statements, whether as a result of new information, future events
or otherwise.
Contact Information:Myesha Lacy
(investors)GBT650-351-4730investor@globalbloodtx.com
Julie Normart (media)W2O
pure415-946-1087media@globalbloodtx.com
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