Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced
the initiation of an early access program for voxelotor, in Europe
and other regions outside the United States, for the treatment of
hemolytic anemia in sickle cell disease (SCD) patients ages 12
years and older.
“With no currently approved therapies outside the United States
to treat hemolytic anemia in sickle cell disease, the unmet need in
this devastating disease is profound,” said Ted W. Love, M.D.,
president and chief executive officer of GBT. “As part of our deep,
long-term commitment to patient access worldwide, we are pleased to
initiate this early access program for eligible patients who may
benefit from voxelotor.”
A first-in-class oral, once-daily therapy, voxelotor directly
inhibits hemoglobin polymerization, the root cause of the sickling
and destruction of red blood cells in SCD. The sickling process
causes hemolytic anemia (low hemoglobin due to red blood cell
destruction), which impairs adequate oxygen delivery to the tissues
and organs in the body.
Voxelotor is approved in the United States under the trade name
Oxbryta® for the treatment of SCD in patients ages 12 years and
older. GBT previously announced it plans to seek regulatory
approval to treat hemolytic anemia in SCD in patients ages 12 years
and older in Europe.
An early access program is a mechanism to make medicines
available pre-approval upon request by a physician for appropriate
patients with no alternative treatment option.
Through the voxelotor early access program, physicians in
countries with an early access regulatory and legal pathway may be
able to request voxelotor for eligible SCD patients who do not have
access to the medicine as part of a clinical trial.
The program is being implemented in partnership with and
administered by Inceptua Group’s Medicines Access division, which
has expertise in the strategy, design, and operational
implementation of pre-approval access programs that make
pharmaceutical products in clinical development available to
patients as appropriate.
All requests must be submitted by the treating physician on
behalf of the eligible SCD patient. Healthcare providers can obtain
details about the voxelotor early access program by contacting
Inceptua at access@inceptua.com.
For the six Middle Eastern countries in the Gulf Cooperation
Council (GCC), GBT previously announced an exclusive
distributorship that references the U.S. approval of Oxbryta to
allow for access to the medicine while health authorities conduct
their regulatory reviews, thereby obviating the need for this new
early access program.
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. With the potential to
improve hemolytic anemia and oxygen delivery, 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.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.
GBT plans to seek regulatory approvals to expand the potential
use of Oxbryta in the United States for the treatment of
SCD in children ages 4 to 11 years and to treat hemolytic anemia in
SCD in patients ages 12 years and older in Europe.
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, 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), 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 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, and vision; the safety, efficacy, and mechanism of
action of Oxbryta and other product characteristics; the
commercialization, delivery, availability, use, and commercial and
medical potential of Oxbryta; ongoing and planned studies of
Oxbryta and related protocols, activities, and expectations; the
potential expansion of the approved use of Oxbryta for more
patients in the U.S., and potential regulatory approval for Oxbryta
to treat patients in Europe; the need for approved therapies
outside the United States to treat hemolytic anemia; an early
access program for voxelotor, including the potential initiation,
framework, availability, use and impact; altering the treatment,
course, and care of SCD and alleviating related complications; the
commitment to patient access to treatment for SCD; the
distributorship for the Gulf Cooperation Council countries,
including access to voxelotor during regulatory review; the
potential of GBT’s pipeline, including inclacumab and other product
candidates; and advancing GBT’s pipeline, 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 the
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 Information: Steven
ImmergutGBT650-410-3258media@gbt.com
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