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
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
'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
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
Prior to potential marketing authorization, GBT initiated an early access program for voxelotor in
About Sickle Cell Disease
SCD 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) 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. 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
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
The EMA has accepted for review GBT's Marketing Authorization Application (MAA) seeking full marketing authorization of Oxbryta in
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 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
About
Forward-Looking Statements
Certain 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
Contact:
Tel: 650-410-3258
Email: simmergut@gbt.com
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