Rebroadcast in sickle cell anemia: voxelotor of GBT company approved

Recently, voxelotor, a drug of global blood therapeutics (GBT), has been approved by the FDA for the treatment of sickle cell anemia (SCD) in adults and adolescents over 12 years old The trade name is oxbryta This is good news again in the field of SCD after the approval of crizanlizumab, a drug of Novartis P-selectin inhibitor With the approval of more and more SCD drugs, patients will have more choices, and the competition in this field will be more intense Sickle cell anemia (SCD) is a kind of hereditary disorder of red blood cells It is named because of the rigidity, viscosity, poor deformability and distortion of red blood cells into "C" or "sickle" shape caused by the defect of hemoglobin in red blood cells Its clinical manifestations are anemia, repeated infection and periodic pain Among them, VASO occlusive crises (VOCs) is one of the most common causes of emergency and hospitalization in patients with SCD, resulting in the decline of quality of life and the increase of risk of death in patients with SCD It is estimated that there are about 100000 SCD patients in the United States with an average life expectancy of 40-60 years, while the average lifetime health care cost per SCD patient is nearly $1 million, and the annual cost of adult patients is more than $30000 Prior to this, the FDA approved SCD treatment drugs were very limited, only hydroxyurea (hydroxyurea), endari (L-glutamine oral powder) and crizanlizumab Among them, hydroxyurea, as an old chemotherapy drug, although the efficacy is significant, can reduce the incidence of vascular obstruction crisis by 50%, but it will cause serious side effects, have a certain risk of cancer, and the use is limited L-Glutamine oral powder was approved by FDA in 2017 to reduce the acute complications of adults and children with SCD aged 5 and above It needs to be taken orally twice a day It is the first drug approved by FDA in recent 20 years to treat SCD Crizanlizumab is a humanized anti-P-selectin monoclonal antibody obtained by Novartis in 2016 when it purchased selexys, an American pharmaceutical company, for us $665 million It has obtained the breakthrough therapy certification, orphan drug qualification and priority evaluation qualification issued by FDA In mid November this year, crizanlizumab was approved by FDA about two months in advance to reduce the frequency of VOC in SCD patients over 16 years old Its trade name is adakveo As the first SCD targeting drug approved by FDA, crizanlizumab will pose a great threat to voxelotor Voxelotor (gbt440) is a small molecule SCD treatment drug that needs to be taken orally once a day It plays a role in the treatment of SCD by increasing the affinity of hemoglobin to oxygen It is found that voxelotor has the potential to improve hemolytic anemia and oxygen transport, and may potentially change the process of SCD Before that, voxelotor was awarded the breakthrough drug qualification, fast track status, orphan drug qualification and rare pediatric disease qualification by FDA In fact, as early as September this year, FDA accepted the new drug application of voxelolor, and designated the target date of PDUFA as February 26, 2020 The approval of voxelotor about three months in advance is based on the positive results of a randomized, double-blind, placebo-controlled phase III study hope The purpose of this study is to evaluate the efficacy and safety of two dose levels of voxelotor (1500mg and 900MG, once daily oral administration) and placebo in the treatment of SCD It has been carried out in 60 research institutions in 22 countries, including 274 cases aged 12 and above SCD patients In the study, patients were randomly divided into 1:1:1 groups and received 1500 mg voxelotor, 900 mg voxelotor or placebo once a day The primary end point was the proportion of patients who achieved hemoglobin response in the intention to treat analysis, which was defined as a more than 1.0g/l increase in hemoglobin relative to baseline at week 24 The final results showed that 59.5% of patients in the 1500 mg treatment group had a hemoglobin level increase of more than 1G / dl, compared with 9.2% in the placebo group See the New England Journal of Medicine (NEJM) for details In terms of safety, the percentage of patients with adverse events or deterioration of adverse events in each experimental group was similar (26% of patients in 1500 mg voxelotor treatment group, 23% in 900 mg voxelotor treatment group, and 26% in placebo group had ≥ Level 3 adverse events) Since then, there are four kinds of SCD treatment drugs approved by FDA However, as a new type of SCD treatment drug, voxelotor and crizanlizumab will form a competition, but who will become the king of this field needs to be verified in time However, it is known that crizanlizumab needs to be injected intravenously once a month, while voxelor needs to be taken orally once a day It is expected that crizanlizumab will have a great advantage in patients' compliance However, crizanlizumab treatment costs a lot Crizanlizumab costs about $85000 to $113000 per year (depending on the dosage) However, the industry is very optimistic about the business prospects of voxelotor According to the report released in June this year by evaluatepharma, a famous pharmaceutical market research institution, voxelotor is expected to become the best-selling SCD drug in the world, and the sales volume in 2024 is expected to reach 1.98 billion US dollars Some analysts have predicted that crizanlizumab's sales in 024 will be about 200 million US dollars GBT is also carrying out a phase IIa study hope-kids1 to evaluate the safety, tolerance, pharmacokinetics and exploratory efficacy of voxelotor in the treatment of children with SCD (4-17 years old) It is expected that voxelotor will become the best-selling drug in the field of SCD in the future [1]FDAapprovesfirsttargetedtherapytotreatpatientswithpainfulcomplicationofsicklecelldisease.RetrievedNovember15,2019