Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT), a
biopharmaceutical company dedicated to the discovery, development
and delivery of life-changing treatments for underserved patient
communities, announced today that the Medicines and Healthcare
Products Regulatory Agency (MHRA) in the United Kingdom has granted
a Promising Innovative Medicine (PIM) designation for voxelotor for
the potential treatment of hemolytic anemia in adults and
adolescent patients 12 years of age and older with sickle cell
disease (SCD).
Following a review by the MHRA, PIM designations are given to
promising treatments that are likely to offer a major advantage for
patients and are an early indication that the treatment is a
promising candidate for the Early Access to Medicines Scheme
(EAMS). For the MHRA to grant a PIM designation, the product must
meet each of the following three criteria:
- The condition should be life-threatening or seriously
debilitating with high unmet need, meaning there is no method of
treatment, diagnosis or prevention available, or existing methods
have serious limitations.
- The medicinal product is likely to offer major advantage over
methods currently used in the UK, based on both non-clinical and
clinical data.
- The potential adverse effects of the medicinal product are
likely to be outweighed by the benefits, allowing for the
reasonable expectation of a positive benefit risk balance.1
SCD is a rare genetic condition which affects approximately
15,000 people in the UK2 and 52,000 people across Europe.3 It has a
devastating impact on the lives of those it affects and their
families, including serious and life-threatening complications that
can lead to organ damage and early death. Despite SCD being the
first genetic disease to be examined at a molecular level,4 there
remains a lack of urgency to treat SCD, which traditionally affects
the African and Caribbean community, and healthcare disparities
based on race persist.
“The sickle cell disease community, which for decades has been
dramatically underserved, deserves innovative treatments that
address the underlying cause of this debilitating disease,” said
Nigel Nicholls, UK general manager of GBT. “Voxelotor is the first
SCD treatment to receive the PIM designation, and this is a
significant milestone in our efforts to potentially make this
therapy available in the UK. This is an important step forward on
our journey, and we remain committed to developing novel treatments
with the hope of transforming the lives of those living with
SCD.”
A first-in-class oral, once-daily therapy, voxelotor directly
inhibits hemoglobin S 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 its plans to seek regulatory
approval from the European Medicines Agency (EMA) for voxelotor in
the treatment of hemolytic anemia in SCD patients ages 12 years and
older.
Prior to potential marketing authorization, GBT initiated an
early access program for voxelotor in Europe and other regions
outside the United States, which enables physicians to use early
access regulatory and legal pathways to request voxelotor for the
treatment of hemolytic anemia in eligible patients with SCD who do
not have access to the medicine as part of a clinical trial. If
approved for EAMS, the voxelotor early access program would be
further extended to eligible UK patients.
About Sickle Cell DiseaseSCD is a rare genetic
condition which affects approximately 15,000 people in the UK2, 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.5 It also affects people
of Hispanic, South Asian, Southern European and Middle Eastern
ancestry.5 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.6 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.6-8 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.7-10
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.11
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. Oxbryta was granted Promising Innovative
Medicine (PIM) designation in Great Britain 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. 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).
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) in the United States, 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 (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 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, mission and vision; safety, efficacy and mechanism of
action of Oxbryta (or voxelotor) 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
voxelotor’s designation as a PIM; ongoing and planned studies 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; the early access program for voxelotor,
including the framework, availability, use and impact; altering the
treatment, course and care of SCD, mitigating related complications
and transforming the lives of people with SCD; 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 activities under GBT’s
collaborative, 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
- ABPI. Early Access to Medicines Scheme.
https://www.abpi.org.uk/media/1329/early_access_to_medicines_scheme.pdf.
Accessed June 1, 2021.
- Sickle Cell Society. About Sickle Cell.
https://www.sicklecellsociety.org/about-sickle-cell/. Accessed June
1, 2021.
- European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Accessed June 12, 2020.
- Smith, T. First molecular explanation of disease. Nat
Struct Mol Biol. 1999:307(6)
- 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:Steven
Immergut (media)650-410-3258simmergut@gbt.com
Courtney
Roberts (investors)650-351-7881croberts@gbt.com
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