Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced
that an expanded access protocol (EAP) for Oxbryta® (voxelotor) in
pediatric patients with sickle cell disease (SCD) has been
initiated and is currently enrolling. The EAP is designed to
provide access to Oxbryta prior to approval for children ages 4 to
11 years with SCD in the United States who have no alternative
treatment options and are ineligible to participate in clinical
trials of Oxbryta. GBT enrolled its first patient in the EAP in
January 2021.
“Early intervention and treatment of sickle cell
disease are critical and have shown potential to modify the course
of this devastating disease. Despite this, children under the age
of 12 have limited approved therapeutic options,” said Ted W. Love,
M.D., president and chief executive officer of GBT. “As part of our
commitment to patient access for Oxbryta, we are pleased to
initiate this expanded access protocol as we work towards our
planned pediatric approval submission for the potential use of
Oxbryta in this age group.”
An oral, once-daily therapy, Oxbryta is the first
and only approved therapy that directly inhibits hemoglobin
polymerization, the root cause of the sickling and destruction of
red blood cells in SCD. Oxbryta is approved in the United States
for the treatment of SCD in patients ages 12 years and older. GBT
previously announced plans to submit a New Drug Application (NDA)
for a pediatric formulation of Oxbryta for the treatment of SCD in
children ages 4 to 11 years by mid-2021.
The EAP allows access to Oxbryta for pediatric
patients 4 to 11 years of age who have an urgent medical need, are
unable to participate in clinical studies, and do not have other
treatment options based on their physician’s judgment. The study
protocol is open-label and includes new age-appropriate
formulations of Oxbryta, including the powder for oral suspension
that will be included in GBT’s planned NDA.
There are multiple clinical sites across the United
States participating in the treatment protocol. For more
information on the location of participating sites, protocol and
eligibility criteria, please visit ClinicalTrials.gov [Identifier:
NCT04724421]. Additional information can be found at the GBT
Medical Information web portal: https://gbtmedinfo.com/ or by
calling 1-833-GBT-4YOU (1-833-428-4968).
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,2 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.3 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.3-5 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.4-7
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, GBT believes Oxbryta blocks
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 Nov.
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.8
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 (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 has been
granted Priority Medicines (PRIME) designation from
the European Medicines Agency (EMA), and
the European Commission (EC) has designated Oxbryta as an
orphan medicinal product for the treatment of patients with
SCD.
The EMA has accepted for review GBT’s Marketing
Authorization Application (MAA) seeking full marketing
authorization of Oxbryta in the European Union to treat hemolytic
anemia in SCD patients ages 12 years and older. GBT also plans to
seek regulatory approval to expand the potential use of Oxbryta
in the United States for the treatment of SCD in children
as young as 4 years old.
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
health care 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 health care 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
health care 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 two weeks after the last dose.
Patients should tell their health care 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
the 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), 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 development to address pain crises
associated with the disease, and GBT021601, 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 treatments for SCD. To learn more, please
visit https://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 and vision; safety, efficacy
and mechanism of action of Oxbryta and other product
characteristics; significance of reducing hemolysis and raising
hemoglobin; commercialization, delivery, availability, use and
commercial and medical potential of Oxbryta; the expanded access
protocol for Oxbryta, including the potential availability,
enrollment, use and impact; ongoing and planned studies of Oxbryta
and related protocols, activities and expectations; regulatory
submissions, review and approval to potentially expand the approved
use of Oxbryta for more patients in the U.S. and to treat
patients in Europe; altering the treatment, course and care of
SCD and mitigating related complications; potential and advancement
of GBT’s pipeline, including inclacumab and other product
candidates; 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 development, manufacture, distribution and commercialization
activities related to Oxbryta; government and third-party payor
actions, including those relating to reimbursement and pricing;
risks and uncertainties relating to competitive products 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 GBT’s and Syros’ research and
development activities under their collaboration; along with those
risks set forth in GBT’s Annual Report on Form 10-K for the fiscal
year ended December 31, 2019, 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.
- 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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