The first highly acidophilic granulocytic syndrome (HES) targeted biopharmaceutical! GlaxoSmithKline Nucala's third indication is reviewed by FDA!

May 28, 2020 /
BiovalleyBIOON/ --GlaxoSmithKline(GSK) recently announced that the U.SFood and Drug Administration (
FDA) has granted it a priority review of a license application for supplementary biological products (sBLA), which seeks approval of the anti-inflammatory drug Nucala (mepolizumab, Mepolizumab) for the treatment of high acidic granulated cell syndrome.) HES is a rare, life-threatening disease caused by eosinophilic inflammation, and current treatment options are limitedif approved, Nucala would be the first targeted biotherapy to treat HES, which would change the treatment pattern for HES patients, and HES would also be Eucala's third indication, which has previously been approved: as an additional maintenance therapy for severe eosinophil-celled granulocytes
asthma(SEA), acidocytocytocytosis granuloma and polyangiotrima (EGPA)Nucala is the first drug to be shown to reduce flares in this rare disease ( symptoms worsen or eosinophil levels above the threshold requires upgraded treatmentIn the United States, Nucala has been granted fast-track and orphan drug status for the treatment of HES before theFDA, and in the European Union, the EMA has granted Nucala therapeutic HES the qualification of orphan drugsthis application, based on positive results from key Phase III clinical studies (NCT02836496) This is a randomized, double-blind, placebo-controlled, 36-week study that assessed the efficacy and safety of Nucala for adolescent and adult treatment for severe HES Severe HES is defined as at least two HES flares in the last 12 months, blood eosinophil count of 1000 cells/microliters In the study, 108 patients were randomly assigned to receive Nucala 300mg (3x100) or a matching placebo every four weeks, while continuing their current HES treatment The main endpoint of the study was the proportion of patients who experienced a HES flare during 32 weeks of treatment, and the secondary endpoints included: the time of the first HES flare (defined as the time of randomization to the occurrence of heS flare), the proportion of patients who experienced a HES flare during the 20th to 32nd week of treatment, the annualization rate of HES flares, and a relative baseline change in the severity of fatigue based on the third assessment of the Simple Fatigue Scale (BFI) results showed that the study reached its main endpoint: during the 32-week period of combined standard care, the proportion of patients with a HES flare in the Nucala treatment group decreased significantly by 50% (56 percent vs 28 percent, p.002) compared to the placebo group The secondary endpoints of the study were also statistically significant and supported the primary endpoint results, with data showing that the risk of the first HES flare in the Nucala treatment group was reduced by 66% (risk ratio of .HR? 0.34,95% CI: 0.1) compared to the placebo group for 32 weeks of treatment 8-0.67), HES flare annualization rate decreased by 66% (incidence ratio ratio of 0.34, 95% CI: 0.19-0.63), fatigue score showed improvement (p.0.036) The security results in this study are consistent with the security known to Nucala Nucala is thought to work by lowering the level of eosinophils in the blood, and there is evidence that the drug has the potential to be a targeted treatment option for a range of inflammatory diseases caused by the growth of eosinophils The data from the key Phase III study above are very encouraging and offer hope to HES patients HES is a rare group of diseases that affect about 20,000 people worldwide and are characterized by the discovery of large numbers of eosinophils in the blood and tissues that, over time, can cause progressive damage to any organ of the body If left untreated, the disease can be fatal HES usually affects the skin, heart, lungs, gastrointestinal tract and central nervous system Symptoms of HES may include coughing, fever, fatigue, asthma
, breathing difficulties, wheezing, repeated upper respiratory tract infections, abdominal pain, vomiting, diarrhea, rashes, arthritis, muscle pain, and joint pain Clinically, heS is treated to reduce eosinophils in the blood and tissues and prevent organ damage and slow progression of the disease, which is typically used in clinical treatments including glucocorticoids, immunomodulating therapy, and cytotoxic therapy Nucala's active pharmaceutical ingredient, mepolizumab, is a monoclonal antibody specifically targeted for leukocyte interleukin 5 (IL-5) IL-5 is a cytokine that regulates the growth, activation, survival of eosinophils (a type of white blood cell) and provides important signals for the migration of eosinophils from bone marrow to the lungs and other organs Nucala binds to human IL-5, blocking the binding of IL-5 to eosinophil surface receptors Inhibiting the binding effect of IL-5 on the receptor in this way can reduce the level of eosinophils in the blood, tissues, and sputum, which in turn reduces the inflammation mediated by eosinophils based on the above mechanism of action, Nucala is being developed for a variety of diseases caused by inflammation caused by eosinophils The drug has been evaluated in 21 clinical trials , more than 3,000 patients, across a variety of eosinophilic metapathy Currently, GSK is evaluating the potential of Nucala to treat HES, nasal polyps, and chronic obstructive pulmonary disease (COPD) Nucala, approved at the end of 2015, is the world's first targeted IL-5 biotherapy To date, Nucala has been approved in the United States, Europe and 20 other markets as an additional maintenance therapy for patients with severe eosinophilic asthma (SEA) In the United States and the European Union, Nucala has also been approved for the treatment of SEA pediatric patients aged 6-17 In addition, Nucala has been approved as an additional maintenance therapy for adult patients with acidophilic granuloma (EGPA) adult patients in the United States, Japan, Canada and other markets (biovalleybioon.com) original source: FDA grants priority review of Nucala for patients with HypereoSinophilic Syndrome (HES)