Global Blood Therapeutics Receives EMA PRIME Designation for GBT440 for the Treatment of Sickle Cell Disease (SCD)
June 28 2017 - 8:00AM
– GBT440 is First Potential Treatment for
SCD To Be Accepted for PRIME –
Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced
that the European Medicines Agency (EMA) has determined that GBT440
for the treatment of sickle cell disease is eligible for its
Priority Medicines (PRIME) program. The PRIME program is a new
regulatory mechanism that provides for early and proactive EMA
support to medicine developers to help patients benefit as early as
possible from innovative new products that have demonstrated the
potential to significantly address an unmet medical need.
“Access to the EMA’s PRIME program is important not
just for GBT, but for the entire SCD community, as this is the
first eligibility granted to PRIME for a SCD therapy. It provides
further recognition from a regulatory authority that SCD is a
severely unmet medical need that requires attention,” said Ted W.
Love, M.D., president and chief executive officer of GBT.
“Additionally, it is external validation of the potential of GBT440
to address this need and bring a major therapeutic advantage to
patients. We look forward to this enhanced collaboration with the
EMA to further accelerate the development of GBT440 for patients in
Europe.”
To be accepted for PRIME, a therapy must
demonstrate the potential to benefit patients with unmet medical
needs through early clinical data. The GBT440 acceptance was
supported by data from the ongoing Phase 1/2 clinical trial
(GBT440-001) evaluating the safety, tolerability, pharmacokinetics
and pharmacodynamics of GBT440 in both healthy subjects and adults
with SCD. Additionally, results from this trial, coupled with the
well understood molecular pathophysiology of SCD and the mechanism
of action of GBT440, support the scientific rationale that
improvement in hemolysis and hemoglobin may be likely to translate
into an improvement in patient symptoms and important disease
modification.
About the Priority Medicines (PRIME)
InitiativePRIME is a program launched by the European
Medicines Agency (EMA) to enhance support for the development of
medicines that target an unmet medical need. This voluntary program
is based on enhanced interaction and early dialogue with developers
of promising medicines, to optimize development plans and speed up
evaluation so these medicines can reach patients earlier. Through
PRIME, the EMA offers early and proactive support to medicine
developers to optimize the generation of robust data on a
medicine’s benefits and risks and enable accelerated assessment of
medicines applications. The goal of the initiative is to help
patients benefit as early as possible from therapies that may
significantly improve their quality of life.
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 GBT440 in Sickle Cell
Disease GBT440 is being developed as an oral, once-daily
therapy for patients with SCD. GBT440 works by increasing
hemoglobin's affinity for oxygen. Since oxygenated sickle
hemoglobin does not polymerize, GBT believes GBT440 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 GBT440 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 GBT440 both Fast Track and Orphan Drug designations for
the treatment of patients with SCD, and the European
Commission (EC) has designated GBT440 as an orphan medicinal
product for the treatment of patients with SCD. GBT is currently
evaluating GBT440 in the HOPE (Hemoglobin Oxygen Affinity
Modulation to Inhibit HbS PolymErization) Study, a Phase 3 clinical
trial in patients age 12 and older with SCD. Additionally, GBT440
is being studied in the ongoing Phase 1/2 GBT440-001 trial and in
the ongoing HOPE-KIDS 1 Study, an open-label, single- and
multiple-dose study in adolescents (age 12 to 17) with SCD designed
to assess the safety, tolerability, pharmacokinetics and
exploratory treatment effect of GBT440.
About Global Blood
Therapeutics Global Blood Therapeutics, Inc. is a
clinical-stage biopharmaceutical company dedicated to discovering,
developing and commercializing novel therapeutics to treat grievous
blood-based disorders with significant unmet need. GBT is
developing its lead product candidate, GBT440, as an oral,
once-daily therapy for sickle cell disease. GBT is also
investigating GBT440 for the treatment of hypoxemic pulmonary
disorders in two ongoing Phase 2a studies in patients with
idiopathic pulmonary fibrosis. To learn more, please
visit www.globalbloodtx.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 GBT440, our ability to implement our clinical development plans
for GBT440 in both SCD and hypoxemic pulmonary disorders, our
ability to generate and report data from our ongoing studies of
GBT440, regulatory review and actions relating to GBT440, 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 regulatory
authorities may disagree with our clinical development plans or
require additional studies or data to support further clinical
investigation of our product candidates, and that drug-related
adverse events may be observed in later stages of clinical
development, 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 March 31, 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.
Myesha Lacy (investors)
GBT
650-351-4730
investor@globalbloodtx.com
Julie Normart (media)
Pure Communications
415-946-1087
media@globalbloodtx.com
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Sep 2024 to Oct 2024
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Oct 2023 to Oct 2024