The new drug for sickle cell disease has been approved by the FDA as a breakthrough drug

. Novartis, the Swiss pharmaceutical giant, recently announced that the U.S. Food and Drug Administration (FDA) has granted Crizanlizumab (SEG101, formerly Known as SelG1) a breakthrough drug qualification (BTD) to prevent vascular obstructive vision (VOC) in all patients with genotype sickle cell disease (SCD).
VOC, also known as sickle cell pain risk (SCPC), is a type of unpredictable and extremely painful event that can lead to severe acute and chronic complications. WHEN multiple blood cells stick together and adhere to blood vessels, VOC occurs, causing blockages. Reducing blood cell and vascular viscosity may help reduce the number of days a patient experiences VOC.
crizanlizumab is an anti-P-selective monoclonal antibody that selectively binds to P-selective protein on the surface of endothell cells and plate plates in blood vessels, leading to the blocking of P-selective. P-selector is the driving factor of vascular astration risk (VOC), a pain complication of SCD. Currently, crizanlizumab is being evaluated for the prevention of VOC in SCD patients, the drug is administered through intravenous infusion once a month, Novarma has planned to file a listing application in the first half of 2019.
the FDA granted crizanlizumab BTD, based on positive data from Phase II SUSTAIN clinical studies. The study, a multi-center, multi-country, randomized, placebo-controlled, double-blind, 12-month study, was designed to assess the efficacy and safety of SCPC in patients with SCD with or without hydroxyurea therapy.
preliminary results of the study, published in the New England Journal of Medicine (NEJM), showed that crizanlizumab significantly reduced the average annual incidence of SCPC by 45.3% (1.63 vs. 2.98, p.010) compared to placebos when using hydroxyurea therapy. The most common adverse reactions in the study (active treatment group (2.5mg/kg, 5mg/kg≥ incidence ≥10%, and twice as much as in the placebo group) included joint pain, diarrhea, itching, vomiting and chest pain. Crizanlizumab treatment did not significantly increase infection.
new post-mortem analysis data released at the 2018 annual meeting of the American Society of Hematology (ASH2018) show that the proportion of patients in the crizanlizumab (5.0mg/kg) treatment group who did not experience any VOC during treatment was significantly higher (37.5% vs 12.2, p. 0.008), the average time to receive the first VOC treatment more than doubled (6.55 months vs 1.58 months, p<0.001), and the annual occurrence rate of VOC decreased (1.04 vs 2.18, p=0.02).
SUSTAIN is part of the SENTRY Clinical Research Project, which currently includes seven clinical studies, with the possibility of adding additional studies to obtain comprehensive data on crizanlizumab for clinical management of SCDs. Several major active therapy studies currently under way include: (1) SOLACE-Adult (A2202) Phase II Study to investigate the pharmacological properties and safety of crizanlizumab in patients aged 16 and over with SCD; Safety and efficacy in pediatric SCD patients; (3) STAND (A2301) Phase III study investigating the efficacy and safety of crizanlizumab in patients 12 years of age and older with SCD; and (4) SUCCESSOR retrospective queue study in adult patients with SCD in the United States.
sickle cell disease (SCD) is a group of inherited red blood cell diseases named after red blood cells that are "C" or "sickle" in shape. SCD patients are prone to vascular obstructive disorder (VOC), especially vascular obstructive pain, which is the main reason SCD patients seek medical care, but there are very few options to prevent VOC. VOC is triggered by clusters of cells that adhere to or block blood flow and are associated with increased morbidity and mortality. Crizanlizumab effectively reduces multicellular adhesion by targeting P-selectives. (Bio Valley)