Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today
announced Oxbryta® (voxelotor) tablets for oral suspension, a new
dispersible, once-daily tablet dosage form recently approved by the
U.S. Food and Drug Administration (FDA), is now available through
GBT’s specialty pharmacy partner network in the United States.
On December 17, 2021, the FDA approved
Oxbryta (voxelotor) tablets for the treatment of sickle cell
disease (SCD) in children ages 4 to less than 12 years – expanding
the previously approved use of Oxbryta to treat SCD in patients
ages 12 years and older in the U.S. The FDA also approved Oxbryta
tablets for oral suspension, the new dispersible tablet dosage form
suitable for patients ages 4 to less than 12 years. 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.
“The sickle cell disease community needs more innovative, safe
and effective treatment options for this disease – a fact central
to GBT’s mission,” said Kim Smith-Whitley, M.D., executive vice
president and head of research and development at GBT. “The FDA’s
recent approval of the new dispersible tablet dosage form of
Oxbryta is an important advance for people and families affected by
this devastating disease, and we are pleased to make this new
dosage form available to patients by prescription. We are grateful
to all those involved in ensuring that more sickle cell disease
patients who need treatment with Oxbryta have access to it.”
Oxbryta is now available by prescription 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 tablets for
oral suspension. The 300 mg dispersible tablets for oral suspension
are available in bottles of 60 and 90 tablets. The dispersible
tablets include grape flavoring and are 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.
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,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.4 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.4-6 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.5-8 Complications of
SCD begin in early childhood and can include neurocognitive
impairment, acute chest syndrome, and silent and overt stroke,
among other serious issues.9
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.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 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
and others; GBT’s commitment to ensuring access to Oxbryta,
including the availability, use and impact of GBT Source Solutions;
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.
- 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.
- Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
- 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.
- Kanter J, et al. Blood Rev. 2013 Nov;27(6):279-87.
- 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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