Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today
announced the U.S. Food and Drug Administration (FDA) has granted
accelerated approval of a supplemental New Drug Application (sNDA)
for Oxbryta® (voxelotor) tablets for the treatment of sickle cell
disease (SCD) in children ages 4 to less than 12 years. This
approval expands the previously approved use of Oxbryta to treat
SCD in patients ages 12 years and older in the United States. The
FDA also approved GBT’s separate New Drug Application (NDA) for
Oxbryta (voxelotor) tablets for oral suspension, a new dispersible,
once-daily tablet dosage form suitable for patients ages 4 to less
than 12 years as well as for older patients who have difficulty
swallowing whole tablets. Oxbryta is the first and only approved
medicine that directly targets sickle hemoglobin polymerization,
the root cause of the sickling and destruction of red blood cells
in SCD.
“For decades, the sickle cell disease community has been
profoundly underserved, and there have been limited treatment
options for younger patients with their whole lives ahead of them.
Complications of SCD that can cause irreversible organ damage are
known to begin in the first few years of life, which is why earlier
intervention is critical,” said Ted W. Love, M.D., president and
chief executive officer of GBT. “Today’s FDA approval of Oxbryta
for children as young as 4 years old – and with a
pediatric-friendly dosage form – is an important advance in the
treatment of this devastating, lifelong condition. GBT is proud to
lead the development of new medicines to address the inadequacies
of care for SCD patients.”
Of the more than 100,000 people in the United States living with
SCD,1 an inherited blood disorder that causes lifelong health
challenges, approximately 16,000 are children between the ages of 4
to 11 years.2 Complications of SCD begin in early childhood and
include neurocognitive impairment, acute chest syndrome, and silent
and overt stroke, among other serious issues.3 In addition, SCD
limits children’s educational attainment and social lives due to
disease complications that often lead to repeated
hospitalizations.4-6 Early intervention and treatment of SCD are
critical and have shown potential to modify the course of this
disease, reduce symptoms and events, prevent long-term organ damage
and extend life expectancy.7
Oxbryta will now be available in two dosage forms for patients 4
years and older based on the patient’s age, weight, and ability to
swallow tablets: 500 mg tablets and 300 mg dispersible
tablets. The dispersible tablet form includes grape flavoring and
is intended to be dispersed in room-temperature clear drinks for
ease of swallowing (such as drinking water or clear soda), making
Oxbryta easier to take for patients who have difficulty swallowing
whole tablets, particularly younger children.
The FDA’s approval of the sNDA is based on data from the
open-label Phase 2a HOPE-KIDS 1 Study (GBT440-007), which showed
that weight-based treatment with the dispersible tablet formulation
of Oxbryta resulted in rapid and sustained improvements in
hemoglobin. Concurrent reduction of hemolysis (or red blood cell
destruction) was also demonstrated.8
“As clinicians, we have had very few approved treatment options
for sickle cell disease and many patients are untreated, which is
very troubling for younger children who may benefit from early
intervention,” said Clark Brown, M.D., Ph.D., director of sickle
cell clinical research at the Aflac Cancer and Blood Disorders
Center of Children’s Healthcare of Atlanta and a primary
investigator of the Phase 2a HOPE-KIDS 1 Study. “This approval is a
significant milestone in the treatment of younger children with SCD
– providing a therapy that addresses the root cause of the disease.
I believe the new dispersible tablets can help patients and their
caregivers achieve daily beneficial treatment and that Oxbryta will
make a meaningful difference in improving the lives of children
living with sickle cell disease.”
“The Sickle Cell Disease Association of America congratulates
GBT for the approval of Oxbryta in children as young as 4 years
old. Having the new formulation and approval down to 4 years of age
is meaningful for families living with sickle cell disease and the
community more broadly,” said Lewis Hsu, M.D., Ph.D., chief medical
officer of the Sickle Cell Disease Association of America. “Due to
the many medical complications associated with sickle cell disease,
kids face challenges every day in their ability to go to school and
have a typical childhood. Adding another new FDA-approved treatment
option for children will enhance care and provide more choices for
children living with sickle cell disease. SCDAA will advocate for
patient-centered decision aids to guide choices. We will work
closely with GBT, our partners, and the sickle cell community to
promote awareness and education about this new therapy to allow
patients to make informed decisions, while advocating for equitable
access to care.”
The FDA instituted its accelerated approval pathway to allow for
earlier approval of drugs that treat serious conditions and that
fill an unmet medical need based on a surrogate endpoint.9 As a
condition of accelerated approval, GBT will continue to study
Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory
study using transcranial Doppler flow velocity to assess the
ability of the therapy to decrease stroke risk in children 2 to 14
years of age. In addition, GBT is continuing to conduct the
HOPE-KIDS 1 Study to demonstrate the benefit of Oxbryta and support
its potential use in children with SCD as young as 9 months of age,
and an ongoing open-label extension study for all patients less
than 18 years of age who have participated in Oxbryta clinical
trials.
GBT is committed to ensuring that patients who are prescribed
Oxbryta can access their medicine. GBT Source Solutions® was
established by GBT as a comprehensive program for patients who are
prescribed Oxbryta that provides a wide range of practical,
educational and financial support customized to each patient’s
needs. GBT Source Solutions provides support by reviewing insurance
coverage options and explaining benefits; explaining specialty
pharmacy benefits and working with the specialty pharmacies that
coordinate shipments of Oxbryta; helping eligible, commercially
insured patients with co-pay assistance; and helping appropriate
patients stay on treatment with a nurse support team. More
information is available at Oxbryta.com or 1-833-428-4968
(1-833-GBT-4YOU).
About Sickle Cell DiseaseSickle cell disease
(SCD) affects more than 100,000 people in the United States,1 an
estimated 52,000 people in Europe,10 and millions of people
throughout the world, particularly among those whose ancestors are
from sub-Saharan Africa.11 It also affects people of Hispanic,
South Asian, Southern European and Middle Eastern ancestry.11 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.12 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.7, 12-13 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 throughout the body. The diminished
oxygen delivery to tissues and organs can lead to life-threatening
complications, including stroke and irreversible organ damage.7,
13-15 Complications of SCD begin in early childhood and can include
neurocognitive impairment, acute chest syndrome, and silent and
overt stroke, among other serious issues.3
About
Oxbryta® (voxelotor) Tablets
and Tablets for Oral SuspensionOxbryta (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 U.S. FDA expanded the approved use of Oxbryta for the
treatment of SCD in patients 4 years of age and older.16
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 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 December 2021, the Committee for Medicinal Products for Human
Use (CHMP) of the EMA adopted a positive opinion recommending
marketing authorization for Oxbryta in Europe for the treatment of
hemolytic anemia due to SCD in adults and pediatric patients 12
years of age and older as monotherapy or in combination with
hydroxycarbamide (hydroxyurea). 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.
Important Safety InformationOxbryta should not
be taken if the patient has had an allergic reaction to voxelotor
or any of the ingredients in Oxbryta. See the end of the patient
leaflet for a list of the ingredients in Oxbryta.Oxbryta can cause
serious side effects, including serious allergic reactions.
Patients should tell their healthcare provider or get emergency
medical help right away if they get rash, hives, shortness of
breath (difficult breathing) or swelling of the face.
The most common side effects of Oxbryta include headache,
diarrhea, stomach-area (abdominal) pain, nausea, rash or hives, and
fever. The most common side effects of Oxbryta in children ages 4
to less than 12 years of age include fever, vomiting, rash,
stomach-area (abdominal) pain, diarrhea, and headache. These are
not all the possible side effects of Oxbryta. Before taking
Oxbryta, patients should tell their healthcare provider about all
medical conditions, including if they have liver problems; if they
are pregnant or plan to become pregnant as it is not known if
Oxbryta can harm an unborn baby; or if they are breastfeeding or
plan to breastfeed as it is not known if Oxbryta can pass into
breastmilk or if it can harm a baby. Patients should not breastfeed
during treatment with Oxbryta and for at least 2 weeks after the
last dose.
Patients should tell their healthcare provider about all the
medicines they take, including prescription and over-the-counter
medicines, vitamins and herbal supplements. Some medicines may
affect how Oxbryta works. Oxbryta may also affect how other
medicines work and may affect the results of certain blood
tests.
Patients are advised to call their doctor for medical advice
about side effects. Side effects can be reported to FDA at
1-800-FDA-1088. Side effects can also be reported to Global
Blood Therapeutics at 1-833-428-4968 (1-833-GBT-4YOU).
Full Prescribing Information for Oxbryta is available
at Oxbryta.com.
About Global Blood TherapeuticsGlobal
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) tablets and tablets for oral
suspension, 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 wave of potential treatments for
SCD. To learn more, please visit www.gbt.com and follow
the company on Twitter @GBT_news.
Forward-Looking StatementsCertain 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; ongoing and planned studies, clinical
trials and registries, and related protocols, activities, timing
and other expectations; significance of the FDA’s approval of GBT’s
regulatory submissions to expand the approved use of Oxbryta for
more patients and in a pediatric formulation in the U.S., including
in advancing the treatment of SCD, the suitability, use and
potential of such pediatric formulation and the impact on patients,
their caregivers and others; impact of having a new treatment
option on the care for children living with SCD, and benefit of
earlier intervention in SCD; GBT’s commitment to ensuring access to
Oxbryta, including the availability, use and impact of GBT Source;
expanding the approved use of Oxbryta to treat patients
in Europe and other territories, including potential
regulatory review, timing and approval; altering the treatment,
course and care of SCD and mitigating related complications;
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.
References
- Centers for Disease Control and Prevention website. Sickle Cell
Disease Research.
https://www.cdc.gov/ncbddd/hemoglobinopathies/scdc-understanding-sickle-cell-disease.html.
Accessed December 1, 2021.
- Symphony Health Claims Data, May 2021.
- Kanter J, et al. Blood Rev. 2013 Nov;27(6):279-87.
- Swanson ME, et al. Am J Prev Med. 2011;41(6S4):S390
–S397).
- Crosby LE, et al. Phys Disabil. 2015;34(1):14-30.
- Smith KE, et al. Dev Neuropsychol. 2016;41(4):231-244.
- Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
- Brown C, et al. Safety and Efficacy of Voxelotor in Pediatric
Patients With Sickle Cell Disease Aged 4-11 Years: Results From the
Phase 2a HOPE-KIDS 1 Study. European Hematology Association
Congress 2021. June 2021.
https://www.gbt.com/wp-content/uploads/2021/06/21-05-19_EHA-007-Oral-Presentation-upload_CBrown.pdf
- Food and Drug Administration website. Accelerated Approval
Program.
www.fda.gov/drugs/information-healthcare-professionals-drugs/accelerated-approval-program.
Accessed November 18, 2021.
- European Medicines
Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Accessed June 12, 2020.
- Centers for Disease Control and Prevention website.
Sickle Cell Disease
(SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html.
Accessed June 3, 2019.
- National Heart, Lung, and Blood Institute website.
Sickle Cell
Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease.
Accessed August 5, 2019.
- Rees DC, et al. Lancet. 2010;376(9757):2018-2031.
- Kato GJ, et al. J Clin Invest.
2017;127(3):750-760.
- Caboot JB, et al. Paediatr Respir Rev.
2014;15(1):17-23.
- Oxbryta (voxelotor) tablets and tablets for oral suspension
prescribing information. South San Francisco, Calif. Global Blood
Therapeutics, Inc.; December 2021.
Contact Information:Steven Immergut (media)+1
650-410-3258simmergut@gbt.com
Courtney Roberts (investors)+1
650-351-7881croberts@gbt.com
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