GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (voxelotor) to Treat Sickle Cell Patients in Europe
Intends to seek full marketing authorization for Oxbryta to treat hemolytic anemia in sickle cell disease
Marketing Authorization Application submission planned by mid-2021
SOUTH SAN FRANCISCO, Calif., June 24, 2020 (GLOBE NEWSWIRE) -- 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 Disease
Sickle 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) Tablets
Oxbryta (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 Information
Oxbryta 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 Therapeutics
Global 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.
- Global Blood Therapeutics, Inc.
- Global Blood Therapeutics, Inc.