Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced
that the U.S. Food and Drug Administration (FDA) has accepted for
filing and review the company’s supplemental New Drug Application
(sNDA) seeking accelerated approval for Oxbryta® (voxelotor) for
the treatment of sickle cell disease (SCD) in children ages 4 to 11
years and its New Drug Application (NDA) seeking approval for a new
age-appropriate dispersible tablet dosage form of Oxbryta suitable
for pediatric patients. Oxbryta, a first-in-class therapy that
directly targets hemoglobin polymerization, the root cause of red
blood cell sickling in SCD, is currently approved by the FDA in a
tablet dosage form to treat SCD in patients age 12 years and older.
The FDA granted Priority Review for both the NDA and sNDA,
providing a six-month review, and assigned a Prescription Drug User
Fee Act (PDUFA) target action date for both applications of
December 25, 2021.
“The FDA’s acceptance of our regulatory submissions for Oxbryta
for the treatment of sickle cell disease in children ages 4 to 11
years and a pediatric-friendly dosage form of Oxbryta is an
important step toward achieving GBT’s goal of bringing Oxbryta to
all eligible patients suffering from this devastating disease,”
said Ted W. Love, M.D., president and CEO of GBT. “There are few
current therapeutic options for children under 12 years of age with
sickle cell disease, which can cause irreversible multi-organ
damage in the first few years of life. Given the profound unmet
need, we appreciate the FDA prioritizing the review of potential
treatments for the long-overlooked sickle cell disease community.”
In the U.S., approximately 17,000 children ages 4 to 11 years have
sickle cell disease.1 The Oxbryta pediatric sNDA and NDA are based
on data from the open-label Phase 2a HOPE-KIDS 1 Study
(GBT440-007). An analysis of data presented at the European
Hematology Association (EHA) 2021 Virtual Congress in 45 children
with SCD ages 4 to 11 years enrolled in the study showed that
weight-based treatment with the Oxbryta dispersible tablets dosage
form that is the subject of the NDA resulted in rapid and sustained
improvements in hemoglobin. Concurrent reduction of hemolysis (or
red blood cell destruction) was also demonstrated.2
The NDA seeks approval for 300 mg dispersible tablets. The
dispersible tablet formulation includes grape flavoring, is
intended to be dispersed in room-temperature drinking water or
other clear drinks for ease of swallowing, and allows for
weight-based dosing in pediatric patients ages 4 to 11 years with
SCD.
Priority review is granted to therapies that the FDA determines
have the potential to provide a significant improvement in the
safety or effectiveness of the treatment, diagnosis or prevention
of a serious condition. Under the PDUFA, a priority review targets
a review time of six months compared to a standard review time of
10 months.
About Sickle Cell DiseaseSickle cell disease
(SCD) affects an estimated 100,000 people in the United
States,1 an estimated 52,000 people in Europe,3 and
millions of people throughout the world, particularly among those
whose ancestors are from sub-Saharan Africa.1 It also affects
people of Hispanic, South Asian, Southern European and Middle
Eastern ancestry.1 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
About
Oxbryta® (voxelotor)
tabletsOxbryta (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, 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. On November 25, 2019, Oxbryta received U.S. Food and
Drug Administration (FDA) accelerated approval for the
treatment of SCD in adults and children 12 years of age and
older.9
As a condition of accelerated approval, GBT is studying 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 15 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 was 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).
The EMA has accepted for review GBT’s Marketing Authorization
Application (MAA) seeking full marketing authorization of Oxbryta
in Europe to treat hemolytic anemia in SCD patients ages 12 years
and older.
Important Safety Information about Oxbryta (voxelotor)
tabletsOxbryta 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, or swelling of the face.
Patients receiving exchange transfusions should talk to their
healthcare provider about possible difficulties with the
interpretation of certain blood tests when taking Oxbryta.
The most common side effects of Oxbryta include headache,
diarrhea, stomach (abdominal) pain, nausea, tiredness, rash, and
fever. 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.
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, the first
FDA-approved treatment that directly inhibits sickle hemoglobin
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 hemoglobin S 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 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 data presented at the EHA Congress, including support for the
use of Oxbryta in children; ongoing and planned studies and
clinical trials and related protocols, activities and expectations;
expanding access to Oxbryta, including related activities and
expectations; significance of the FDA’s acceptance of GBT’s
regulatory submissions for Oxbryta; regulatory submissions to
potentially expand the approved use of Oxbryta for more patients
and in a pediatric formulation in the U.S. and to treat
patients in Europe and other territories, including potential
review, timing and approval; the suitability, use and potential of
such pediatric formulation; altering the treatment, course and care
of SCD and mitigating related complications; potential impact of
addressing the root cause of red blood cell sickling at a young
age; 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
(SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html.
Accessed June 3, 2019.
- 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
- European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Accessed June 12, 2020.
- 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. Nat Rev Dis Primers. 2018;4:18010.
- 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 prescribing information. South San
Francisco, Calif. Global Blood Therapeutics, Inc.; November
2019.
Contact:Steven
Immergut (media)650-410-3258simmergut@gbt.com
Courtney
Roberts (investors)650-351-7881croberts@gbt.com
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