Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) announced
today it has received the 2020 Rare Impact Award® for Industry
Innovation from the National Organization for Rare Disorders
(NORD). GBT received the award for Oxbryta® (voxelotor) tablets at
a virtual event on October 9, 2020, following NORD’s annual Rare
Disease and Orphan Products Breakthrough Summit.
Discovered and developed by GBT, 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 sickle
cell disease (SCD). It is currently approved in the United
States to treat SCD in adults and adolescents age 12 years and
older.
“We are honored to receive this recognition from NORD for
developing novel therapeutics that meet the needs of the sickle
cell community, which has been underserved for far too long,” said
Ted W. Love, M.D., president and CEO of GBT. “The true winners are
sickle cell disease patients and their families, who now have more
available treatment options. We look forward to continuing to work
closely with healthcare providers, patients, and the rare disease
community, whose partnership was integral to the development of
Oxbryta, to advance our shared goal of making a difference in the
lives of people with this devastating, inherited disease.”
The NORD Rare Impact Awards celebrate the individuals, groups,
and companies making extraordinary contributions to the lives of
rare disease patients and caregivers. NORD is the leading
independent advocacy organization committed to the identification,
treatment, and cure of rare disorders through programs of
education, advocacy, research, and patient services.
In addition, on September 24, 2020, Oxbryta was selected by the
2020 National Xconomy Awards as Breakthrough Drug of the Year. Each
year, the National Xconomy Awards honor the top individuals,
companies, and organizations within the United States life sciences
ecosystem.
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 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, 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.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, Oxbryta inhibits sickle
hemoglobin polymerization and the resultant sickling and
destruction of red blood cells. 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 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
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 Oxbryta in its Priority Medicines (PRIME) program, 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 age 4 to 11 years and to treat hemolytic anemia in SCD in
people age 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 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. 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.
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 650-410-3258simmergut@gbt.com
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