Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today
announced plans to expand the potential use of Oxbryta® (voxelotor)
for the treatment of sickle cell disease (SCD) in children ages 4
to 11 years, following a Type B meeting with the U.S. Food and Drug
Administration (FDA).
Oxbryta is the first and only therapy that directly inhibits
hemoglobin polymerization, the root cause of the sickling and
destruction of red blood cells in SCD. It is currently approved in
the United States to treat SCD in adults and adolescents ages 12
years and older.
“Sickle cell disease is a devastating, lifelong inherited
condition marked by hemoglobin polymerization, which occurs in
every person with the disease regardless of age. By mitigating red
blood cell sickling and destruction early in life, we hope to
positively impact the course of the disease and alleviate the
serious and life-threatening complications that older individuals
with sickle cell disease often suffer,” said Ted W. Love, M.D.,
president and chief executive officer of GBT. “We are pleased to
have agreed with the FDA on a potential path forward to expand the
use of Oxbryta in younger children, and we look forward to
submitting a New Drug Application by the middle of next year.”
GBT intends to submit a New Drug Application (NDA) for Oxbryta,
which will include a new age-appropriate formulation, for the
treatment of SCD in children ages 4 to 11 years under the FDA’s
accelerated approval pathway. The NDA will include clinical data
from the ongoing Phase 2a HOPE-KIDS 1 study. The FDA’s accelerated
approval pathway can allow for earlier approval of drugs that treat
serious conditions and that fill an unmet medical need based on a
surrogate endpoint.
About Sickle Cell DiseaseSickle cell disease
(SCD) affects an estimated 100,000 people in the United States and
millions of people throughout the world, particularly among those
whose ancestors are from sub-Saharan Africa. It also affects people
of Hispanic, South Asian, Southern European and Middle Eastern
ancestry.1 SCD is a lifelong inherited blood disorder that impacts
hemoglobin, a protein carried by red blood cells that delivers
oxygen to tissues and organs throughout the body.2 Due to a genetic
mutation, people 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.5-7
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.7 As a condition of accelerated approval, GBT will
continue to study voxelotor in the HOPE-KIDS 2 Study, a
post-approval confirmatory study using transcranial Doppler (TCD)
flow velocity to assess the ability of Oxbryta 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. The European Medicines Agency (EMA)
has included voxelotor in its Priority Medicines (PRIME) program,
and the European Commission (EC) has designated voxelotor
as an orphan medicinal product for the treatment of patients with
SCD.
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. In addition, GBT’s drug discovery teams are working on new
targets to develop the next generation 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, 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
labeling for Oxbryta, including associated plans, activities,
timing and expectations; the NDA for such expansion, including its
contents and submission, the pathway for potential approval and FDA
review; altering the treatment, course and care of SCD and
alleviating related complications; the potential of inclacumab; 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 has only recently established its commercialization
capabilities and may not be able to successfully commercialize
Oxbryta; risks associated with GBT’s dependence on third parties
for development, manufacture 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 funding and other
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.
- 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 Immergut
(media)650-410-3258simmergut@gbt.com
Stephanie Yao (investors)650-741-7730syao@gbt.com
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