Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) announced
today that Oxbryta® (voxelotor) tablets, a first-in-class oral
therapy indicated for the treatment of sickle cell disease (SCD) in
patients age 12 years and older in the United States, has been
awarded the prestigious 2021 Prix Galien USA Award for Best
Biotechnology Product. The Prix Galien award is among the global
health industry's most renowned honors, recognizing outstanding
product achievements that improve the human condition. Oxbryta was
selected among 19 nominees by a committee of 12 leaders from the
biotech industry and academia, including five Nobel Laureates.
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.
“The Prix Galien Foundation’s recognition of Oxbryta is a
testament to the importance of this innovation for a patient
community that has suffered from limited treatment options for far
too long. With the potential to modify the course of this disease,
we are proud that Oxbryta is able to help many people with SCD
today and in the future,” said Ted W. Love, M.D., president and CEO
of GBT. “Receiving the prestigious Prix Galien Award is a
tremendous honor. We are grateful to all the patients, their
families, and physicians, as well as the entire GBT team for their
tireless dedication in making this recognition possible.”
Oxbryta has previously been recognized with the 2020 National
Xconomy Award for Breakthrough Drug of the Year and the 2020
National Organization for Rare Disease (NORD) Rare Impact Award for
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
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, 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 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
As a condition of accelerated approval, GBT is studying 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
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 was granted Priority
Medicines (PRIME) designation from the European Medicines Agency
(EMA), Oxbryta was designated by the European Commission (EC) as an
orphan medicinal product for the treatment of patients with SCD,
and Oxbryta was granted Promising Innovative Medicine (PIM)
designation in the United Kingdom 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 ia also seeking 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 Information about Oxbryta (voxelotor)
tabletsOxbryta 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
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
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) tablets, 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 Phase 3 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 potential treatments for SCD. To learn
more, please visit www.gbt.com and follow the company on
- Centers for Disease Control and Prevention website. Sickle
Accessed June 3, 2019.
- European Medicines Agency.
Accessed June 12, 2020.
- National Heart, Lung, and Blood Institute website.
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.
- Caboot JB, et al. Paediatr Respir Rev.
- Oxbryta (voxelotor) tablets prescribing information. South San
Francisco, Calif. Global Blood Therapeutics, Inc.; November
Contact:Steven Immergut (media)+1
Courtney Roberts (investors)+1
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Dec 2021 to Jan 2022
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Jan 2021 to Jan 2022