Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today
announced the European Commission (EC) has granted Marketing
Authorization for Oxbryta® (voxelotor) for the treatment of
hemolytic anemia due to sickle cell disease (SCD) in adult and
pediatric patients 12 years of age and older as monotherapy or in
combination with hydroxycarbamide (hydroxyurea). Oxbryta, a
once-daily, oral treatment, is the first medicine approved in
Europe that directly inhibits sickle hemoglobin (HbS)
polymerization, the molecular basis of sickling and destruction of
red blood cells in SCD.
“Living with sickle cell disease is a challenging life-long
journey for patients and their families and can negatively impact
every aspect of their lives. At GBT, our goal is to transform the
treatment of sickle cell disease, and we are immensely proud to
have developed an innovative medicine that we believe has the
potential to change the trajectory of the disease,” said Sebastian
Stachowiak, head of Europe and GCC at GBT. “We are thankful to the
investigators, patients and others in the sickle cell community who
supported the development of this therapy, and we hope it will make
a very significant difference to the thousands of people in Europe
living with the debilitating impact of this disease.”
SCD is one of the most prevalent genetic diseases in Europe,
affecting approximately 52,000 people,1 primarily of African,
Mediterranean and South Asian descent.2 In SCD, red blood cells
lose their flexibility and become rigid, sticky and crescent or
sickle shaped. The sickling process causes the breakdown of red
blood cells (hemolysis) and consequent hemolytic anemia (low
hemoglobin and low oxygen delivery in the body due to red blood
cell destruction), which is experienced in varying degrees by all
those living with SCD.3 Vasculopathy (lesions in small blood
vessels) is caused by hemolysis and in conjunction with sickled
cells, promotes blockages in capillaries and small blood vessels,
causing pain and impeding the flow of blood and supply of oxygen
throughout the body. People living with SCD experience
progressive and life-threatening complications and morbidities,
including damage to major organs such as the liver, kidneys, lungs
and heart – which contribute to decreased quality of life and early
death.4
“Hemolytic anemia is the root cause of significant morbidity in
people living with sickle cell disease, yet available treatment
options are limited,” said Dr. Baba Inusa, consultant and professor
of paediatric haematology, Guy’s and St. Thomas’ NHS Foundation
Trust, London and chair of the National Haemoglobinopathy Panel in
England. “Treatment with Oxbryta has been shown to break the
recurrent cycle of red blood cell sickling leading to their
destruction – significantly improving patients’ hemoglobin levels
and the blood’s oxygen-carrying capacity.”
The EC approval follows a positive opinion by the Committee for
Medicinal Products for Human Use (CHMP) in December 2021 based on
results of the Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation
to Inhibit HbS PolymErization) Study demonstrating clinically
meaningful and statistically significant improvements in hemoglobin
(Hb) levels, accompanied by hemolysis, for patients treated with
Oxbryta. Results from the HOPE Study were published in June 2019 in
The New England Journal of Medicine and the analysis of the
complete data from the HOPE Study were published in The Lancet
Haematology in April 2021.
In the HOPE Study, Oxbryta demonstrated a favorable safety
profile with limited and transitory adverse reactions. The most
common adverse reactions occurring in ≥10% of patients treated with
Oxbryta (1500mg) with a difference of >3% compared to placebo
were headache (26% vs. 22%), diarrhea (20% vs. 10%), abdominal pain
(14% vs. 8%), nausea (17% vs. 10%), fatigue (14% vs. 10%), rash
(14% vs. 10%) and pyrexia (12% vs. 7%).5
The EC approval means Oxbryta has received marketing
authorization in all EU member states, as well as in Iceland,
Liechtenstein and Norway. In the United Kingdom, GBT has submitted
an application to the Medicines and Healthcare products Regulatory
Agency (MHRA) for a Great Britain Marketing Authorisation using the
EC Decision Reliance Procedure.
About Sickle Cell Disease Sickle cell
disease (SCD) affects more than 100,000 people in the United
States,3 an estimated 52,000 people in Europe,2 and millions of
people throughout the world, particularly among those whose
ancestors are from sub-Saharan Africa.3 It also affects people of
Hispanic, South Asian, Southern European and Middle Eastern
ancestry.3 SCD is a lifelong inherited rare blood disorder that
impacts hemoglobin, a protein carried by red blood cells that
delivers oxygen to tissues and organs throughout the body.6 Due to
a genetic mutation, individuals with SCD form abnormal hemoglobin
known as sickle hemoglobin. Through a process called hemoglobin
polymerization, red blood cells become sickled – deoxygenated,
crescent-shaped and rigid.3, 6, 7 The sickling process causes
hemolytic anemia (low hemoglobin due to red blood cell destruction)
and blockages in capillaries and small blood vessels, which impede
the flow of blood and oxygen delivery throughout the body. The
diminished oxygen delivery to tissues and organs can lead to
life-threatening complications, including stroke and irreversible
organ damage.4, 7, 8, 9 Complications of SCD begin in early
childhood and can include neurocognitive impairment, acute chest
syndrome, and silent and overt stroke, among other serious
issues.10
About
Oxbryta® (voxelotor)
Oxbryta (voxelotor) is an oral, once-daily therapy for patients
with sickle cell disease (SCD). Oxbryta works by increasing
hemoglobin’s affinity for oxygen. Since oxygenated sickle
hemoglobin does not polymerize, Oxbryta inhibits sickle hemoglobin
polymerization and the resultant sickling and destruction of red
blood cells leading to hemolysis and hemolytic anemia, which are
primary pathologies faced by every single person living with SCD.
Through addressing hemolytic anemia and improving oxygen delivery
throughout the body, GBT believes that Oxbryta has the potential to
modify the course of SCD.
In November 2019, the U.S. Food and Drug
Administration (FDA) granted accelerated approval for Oxbryta
tablets for the treatment of SCD in adults and children 12 years of
age and older, and in December 2021, the FDA expanded the
approved use of Oxbryta for the treatment of SCD in patients 4
years of age and older in the United States.10 As a condition of
accelerated approval for patients ages 4 and older in the
United States, GBT will continue to study Oxbryta in the HOPE-KIDS
2 Study, a post-approval confirmatory study using transcranial
Doppler (TCD) flow velocity to assess the ability of the therapy to
decrease stroke risk in children 2 to 14 years of age.
In recognition of the critical need for new SCD treatments, the
FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug,
and Rare Pediatric Disease designations for the treatment of
patients with SCD. Additionally, Oxbryta received the prestigious
2021 Prix Galien USA award for “Best Biotechnology Product” from
The Galien Foundation.
Oxbryta has been granted Priority Medicines (PRIME) designation
from the European Medicines Agency (EMA), Oxbryta was
designated by the European Commission (EC) as an orphan
medicinal product for the treatment of patients with SCD, and
Oxbryta was granted Promising Innovative Medicine (PIM) designation
in the United Kingdom from the Medicines and Healthcare
products Regulatory Agency (MHRA). In addition, the Ministry
of Health and Prevention (MOHAP) in the United Arab
Emirates (UAE) has granted marketing authorization for Oxbryta
for the treatment of SCD in adults and children 12 years of age and
older.
Please click here for Important Safety Information and full
Prescribing Information including Patient Information for Oxbryta
in the U.S.
About Global Blood Therapeutics Global
Blood Therapeutics (GBT) is a biopharmaceutical company dedicated
to the discovery, development and delivery of life-changing
treatments that provide hope to underserved patient communities.
Founded in 2011, GBT is delivering on its goal to transform the
treatment and care of sickle cell disease (SCD), a lifelong,
devastating inherited blood disorder. The company has introduced
Oxbryta® (voxelotor), the first FDA-approved medicine that directly
inhibits sickle hemoglobin (HbS) polymerization, the root cause of
red blood cell sickling in SCD. GBT is also advancing its pipeline
program in SCD with inclacumab, a P-selectin inhibitor in Phase 3
development to address pain crises associated with the disease, and
GBT021601 (GBT601), the company’s next generation HbS
polymerization inhibitor. In addition, GBT’s drug discovery teams
are working on new targets to develop the next generation of
treatments for SCD. To learn more, please visit www.gbt.com and
follow the company on Twitter @GBT_news.
Forward-Looking Statements Certain
statements in this press release are forward-looking within the
meaning of the Private Securities Litigation Reform Act of 1995,
including statements containing the words “will,” “anticipates,”
“plans,” “believes,” “forecast,” “estimates,” “expects” and
“intends,” or similar expressions. These forward-looking statements
are based on GBT’s current expectations and actual results could
differ materially. Statements 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. GBT intends these forward-looking
statements, including statements regarding GBT’s priorities,
dedication, commitment, focus, goals, mission and vision; safety,
efficacy and mechanism of action of Oxbryta and other product
characteristics; significance of reducing sickling and hemolysis
and raising hemoglobin; commercialization, delivery, availability,
use and commercial and medical potential of Oxbryta; significance
of the Marketing Authorization for Oxbryta by the EC; ongoing and
planned studies, clinical trials and registries and related
protocols, activities, timing and other expectations; expanding the
approved use of Oxbryta, including related activities and
expectations; altering the treatment, course, trajectory and care
of SCD, mitigating related complications and making a difference to
people living with SCD; safety, efficacy, mechanism of action,
advancement and potential of GBT’s drug candidates and pipeline;
and working on new targets and discovering, developing and
delivering treatments, 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
GBT makes this statement for purposes of complying with those safe
harbor provisions. These forward-looking statements reflect GBT’s
current views about its plans, intentions, expectations, strategies
and prospects, which are based on the information currently
available to the company and on assumptions the company has made.
GBT 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 GBT’s control, including, without
limitation, risks and uncertainties relating to the COVID-19
pandemic, including the extent and duration of the impact on GBT’s
business, including commercialization activities, regulatory
efforts, research and development, corporate development activities
and operating results, which will depend on future developments
that are highly uncertain and cannot be accurately predicted, such
as the ultimate duration of the pandemic, travel restrictions,
quarantines, social distancing and business closure requirements in
the U.S. and in other countries, and the effectiveness of actions
taken globally to contain and treat the disease; the risks that GBT
is continuing to establish its commercialization capabilities and
may not be able to successfully commercialize Oxbryta; risks
associated with GBT’s dependence on third parties for research,
development, manufacture, distribution and commercialization
activities; government and third-party payer actions, including
those relating to reimbursement and pricing; risks and
uncertainties relating to competitive treatments and other changes
that may limit demand for Oxbryta; the risks regulatory authorities
may require additional studies or data to support continued
commercialization of Oxbryta; the risks that drug-related adverse
events may be observed during commercialization or clinical
development; data and results may not meet regulatory requirements
or otherwise be sufficient for further development, regulatory
review or approval; compliance with obligations under the Pharmakon
loan; and the timing and progress of activities under GBT’s
collaboration, license and distribution agreements; along with
those risks set forth in GBT’s Annual Report on Form 10-K for the
fiscal year ended December 31, 2020, and in GBT’s most recent
Quarterly Report on Form 10-Q filed with the U.S. Securities and
Exchange Commission, as well as discussions of potential risks,
uncertainties and other important factors in GBT’s subsequent
filings with the U.S. Securities and Exchange Commission. Except as
required by law, GBT assumes no obligation to update publicly any
forward-looking statements, whether as a result of new information,
future events or otherwise.
Contact:
Steven Immergut (media)+1 650 410 3258simmergut@gbt.com
Claudia Nabaie (media Europe)+41 79 906 5814cnabaie@gbt.com
Courtney Roberts (investors)+1 650 351 7881croberts@gbt.com
References
1 European Medicines Agency. EU/3/18/2125: Orphan designation
for the treatment of sickle cell disease.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Last accessed February 2022.2 Centers for Disease Control and
Prevention website. Sickle Cell Disease (SCD).
https://www.cdc.gov/ncbddd/sicklecell/data.html. Last accessed
February 2022.3 European Medicines Agency. New treatment for sickle
cell disease.
https://www.ema.europa.eu/en/news/new-treatment-sickle-cell-disease.
Last accessed February 2022. 4 Kato GJ et al. Nat Rev Dis Primers.
2018;4:18010.5 Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V,
El-Beshlawy A, et al. A phase 3 randomized trial of Voxelotor in
sickle cell disease. N Engl J Med. 2019;381(6):509–19.6 National
Heart, Lung, and Blood Institute website. Sickle Cell Disease.
https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Last
accessed February 2022.7 Rees DC, et al. Sickle cell disease.
Lancet. 2010;376(9757):2018-2031.8 Kato GJ et al. Intravascular
hemolysis and the pathophysiology of sickle cell disease. J Clin
Invest. 2017;127:750-760.9 Caboot JB, et al. Hypoxemia in sickle
cell disease: significance and management Paediatr Respir Rev.
2014;15(1):17-23.10 Cooper TE et al. Pharmacological interventions
for painful sickle cell vaso-occlusive crises in adults. Cochrane
Database of Systematic Reviews. 2019.
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