Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today
announced that it plans to submit a Marketing Authorization
Application (MAA) to the European Medicines Agency (EMA) for
Oxbryta® (voxelotor) tablets to treat hemolytic anemia in
sickle cell disease (SCD) patients ages 12 years and older. The
company plans to submit the MAA by mid-2021.
An oral, once daily therapy, Oxbryta 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.
“Anemia and hemolysis are well established to cause morbidity
and mortality in sickle cell disease. At GBT, our focus is to
address sickle cell disease at its core and modify the course of
the disease, with the goal of mitigating the serious and
life-threatening complications that often lead to long-term damage
and early death,” said Ted W. Love, M.D., president and chief
executive officer of GBT. “Currently, there are no approved
therapies in Europe to treat hemolytic anemia in sickle cell
disease. We hope to address this critical unmet need as part of our
commitment to patient access to innovative treatments for this
devastating disease.”
Based on recent discussions with the EMA, GBT intends to seek
full marketing authorization of Oxbryta. The planned MAA will
include data from the Phase 3 HOPE (Hemoglobin Oxygen Affinity
Modulation to Inhibit HbS PolymErization) Study and the Phase 2
HOPE-KIDS 1 Study, both of which enrolled patients at clinical
sites in Europe. GBT also intends to initiate an Early Access
Program in Europe for patients and physicians who may need access
to Oxbryta prior to potential marketing authorization.
In Europe, an estimated 52,000 people are living with SCD.1 In
recognition of the need for new SCD treatments, the EMA granted
Oxbryta Priority Medicines (PRIME) designation, and the European
Commission (EC) designated Oxbryta as an orphan medicinal product
for the treatment of patients with SCD.
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 on November 25, 2019.2 Earlier
this month, GBT announced plans to expand the potential use of
Oxbryta in the United States for the treatment of SCD in children
ages 4 to 11 years.
About Sickle Cell DiseaseSickle cell disease
(SCD) affects an estimated 100,000 people in the United States,3 an
estimated 52,000 people in Europe,1 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 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, 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.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, 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.2
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.
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 regulatory
approval for Oxbryta to treat patients in Europe, including the
submission of an MAA, the type and contents of such submission and
related timing; addressing the need for approved therapies in
Europe to treat hemolytic anemia; an Early Access Program in Europe
for Oxbryta; the potential expansion of the labeling for Oxbryta in
the US; altering the treatment, course, and care of SCD and
alleviating related complications; the commitment to patient access
to treatments for SCD; 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
- European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Accessed June 12, 2020.
- Oxbryta (voxelotor) tablets prescribing
information. South San Francisco, Calif. Global Blood
Therapeutics, Inc.; November 2019.
- 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.
Contact Information:Steven Immergut
(media)650-410-3258simmergut@gbt.com
Stephanie Yao (investors)650-741-7730syao@gbt.com
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Aug 2024 to Sep 2024
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Sep 2023 to Sep 2024