November New Drug Approval Profile: 8 U.S. China 2 EU 1 (above)

01Air polymer-type AAirpolymer-type A has been approved by the FDA on November 7, 2019 for ultrasound of the uterine fallopian tube sinopia to assess the fluidity of the fallopian tubes of women who are known or suspected of infertilityThe drug was developed by GISKIT B.Vand is called ExEm®couples are infertile, about 12 to 33% of cases are caused by blockages in the fallopian tubesTherefore, assessing the smoothness of the fallopian tubes is a routine measure for detecting infertilityExEm ® is composed of hydroxyelycellin, an echocontrast agentWhen visualized using ultrasound, the foam displays an echo or bright signal in the fallopian tubes and peritoneal cavityExEm ® was approved based on two hyFoSy studies of the uterine fallopian tube ultrasound foamIn Test A, 2D imaging was able to assess 80% of fallopian tube obstruction and 92% of the fallopian tubes were clear, and 3D imaging tests reached 98% and 91%, respectivelyTest B combined 2D/3D imaging technology, the test results are similar to test A02LuspaterceptLuspatercept-aamt has been approved by the FDA on November 8, 2019 for the treatment of beta-ground poverty that requires regular red blood cell transplants, named Reblozyl ®The drug was originally developed by Acceleron and later licensed to The New Basenormal human hemoglobin is made up of 2 alpha beglodoproteins and 2 beta-gloaffers Beta-ground poverty is due to defects or mutations in the genes that encode beta-gloper, the amount of beta-gloper synthesis decreases, and alpha-gloper suplodoin is excessive and induces red blood cell apoptosis in the form of enclustosis The worldwide incidence of the disease is 1/100,000, in the European Union 1/10,000, while Cyprus (incidence 14 per cent), Sardinia (10.3 per cent), South-East Asia and other countries and regions are the most serious At present, long-term blood transfusion combined iron chelating agent, hematopoietic stem cell transplantation is the main treatment plan, but there are iron deposition and HLA matching difficulties and other shortcomings Reblozyl ® is a recombinant fusion protein that targets the TGF-beta family ligand sympathis, which can reduce smad2/3 signaling pathways The drug enhances the growth capacity of red blood cells by promoting the differentiation of late precursor red blood cells Reblozyl ® was approved based on a multicenter, randomized, double-blind, placebo-controlled trial (NCT02604433) 336 patients in poverty were given Reblozyl ® or placebo at 2:1 The blood transfusion in 21% of the treatment group decreased by at least 33%, while only 4.5% of patients in the placebo group met the target (p 0.0001) Reblozyl ® is the first red blood cell-mature drug approved in the U.S to ease patients' dependence on blood transfusions 03
Cefiderocol Sulfate Tosylate Cefiderocol, developed by Yannoyi Pharmaceuticals, was approved by the FDA on November 14, 2019 for the treatment of complex urinary tract infections (cUTI) under the name Ofroja® In addition, the drug was submitted to the EU EMA in April 2019 cUTI common pathogenic bacteria include E coli, Krebs, pseudomonas and other gram-negative bacteria, enterococci and other parts of gram-positive bacteria, mainly manifested in the structure and function of the genitourinary system abnormal Wide-spectrum antibiotics, surgery and catheterization are often used clinically to treat the treatment Cefiderocol is a cephalosporin antibiotic drug against Gram-negative bacteria and is used as an iron carrier to adsorbed extracellular free iron Cefiderocol can passively diffuse through the pore protein or through an active transport mechanism of an iron carrier to enter the peripheral gap of bacteria Cefiderocol inhibits the biosynthesis of bacterial cell walls by binding to the penicillin-binding protein family (PBPs) Cefiderocol was approved on the basis of an international multi-center, randomized, double-blind trial (NCT02321800), in which 448 cUTI inpatients received cefideroero or colpenem/cilastatin treatment at 2:1 The results showed that on the seventh day of treatment, 72.6% of patients in the cefiderocol group achieved bacterial removal and clinical remission, compared with 54.6% in the control group high mortality rate from cUTI caused by Gram-negative bacteria is a medical challenge, and Yannoi believes The Fetroja ® will be one of the best drug treatment options 04
Zebutini developed by Zanubruminib Baiji Shenzhou was approved by the FDA on November 14, 2019 for the treatment of adult set cell lymphoma (MCL) under the name BrukinsaTM MCL is a B-cell non-Hodgkin lymphoma, the main disease population is middle-aged and elderly MCL begins with lymph nodes and gradually spreads to the spleen, bone marrow, blood, and even the esophagus, gastrointestinal and other areas ACCORDING TO THE AMERICAN CANCER SOCIETY, MCL ACCOUNTS FOR ABOUT 5% OF LYMPHOMA BrukinsaTM is a small molecule BTK (Bruton tyrosine kinase) inhibitor that blocks the transmission of associated signals, thereby inhibiting the growth of malignant proliferation B cells and killing tumor cells BrukinsaTM's approval was based on a clinical phase II, open label, multi-center, single-arm trial (NCT03206970) that included 86 MCL patients who had been treated in the past The results showed that 84% of patients achieved tumor reduction, with a median total remission period of 19.5 months Another one-arm trial (NCT02343120) included 32 patients, 84% of the patients had a reduced tumor block, and the median total remission period was 18.5 months BrukinsaTM not only broke the side effects of ibitinib on other targets, but also significantly improved drug tolerance concentrations China has implemented drug reform since 2015, which has increased the enthusiasm of a large number of domestic pharmaceutical companies for research and development Based on the favorable environment of domestic policy, BrukinsaTM became the first Chinese original research drug to go overseas 05
Crizanlizumab Crizanlizumab was approved by the FDA on November 15, 2019, and the drug was developed by Selexys Pharma (acquired by Novartis in 2012) to treat vascular occlusion (VOCs) in patients with sickle cell disease, named Adakveo® sickle cell anemia is a complex, exhaustive blood genetic disease that is not limited to red blood cells Clinical manifestations of chronic inflammation, P-selectin and other cell adhesion protein increase, cell coagulation, blood flow blockage, leading to vascular clotted acetic elephant (VOCs) and other life-threatening complications VOCs are the main cause of hospitalization for patients with sickle cell anemia, with approximately 200,000 CASEs of ER emergency in the United States each year Adakveo ® is a humanized IgG2 kappa monoclonal antibody that targets P-selectin, which can block the interaction between P-selectors and their ligands such as PSGL-1 Adakveo ® inhibits the coagulation of endothelial, red blood cells, and white blood cells by binding to P-selectins in activated endothelial cells and platelets Adakveo ® was approved based on a SUSTAIN clinical study (NCT01895361) The median annual incidence of VOC was lower in the treatment group compared to the placebo group (1.63 vs 2.98, p 0.010) About 36 percent of patients in the treatment group had VOC events, compared with 17 percent in the placebo group The median time of first VOC in the treatment group and 1.4 months in the placebo group was 4.1 months and 1.4 months, respectively Adakveo ® is the first monoantigen to target P-selectin, which is instructive for drug development in the field of SCD 06
Givosiran Alnylam developed givosiran has been approved by the FDA on November 20, 2019 for the treatment of adult acute hepatic rickets (AHP), named Givlaari ® AHP is an extremely rare genetic disease that endangers the life and quality of life of patients There are four types of AHP: acute intermittent rickets (AIP), hereditary fecal dysentery (HCP), polysaccosis (VP), and autosomal recessive inheritance of 5-aminoacetic acetaminophen dehydrated acetaminophen dehydrated acetaminophen deficiency rickets (ADP) The overall incidence of AHP is 5/100,000, and the genetic excitation rate of AIP, HCP and VP is very low, and more than 90% of hybrids carrying pathogenic mutations can survive asymptomaticly Givlaari ® is a 5-amino acetyl propylene acetaminophenase 1 (ALAS1)-oriented small interfering RNA that can reduce elevated ALAS1 mRNA levels in liver cells by means of RNA interference Givlaari ® was approved based on a randomized, double-blind, placebo-controlled international trial (NCT03338816) 94 patients were included in the AHP group, which entered the treatment group or placebo group according to 1:1 Givlaari ® group had a 70% reduction in seizures compared to a placebo RNAi technology was named science magazine's top 10 technology advances in 2001 In August 2018, Alnylam launched the world's first RNAi drug, patisiran, while givosiran's approval and other drugs under study demonstrated Alnylam's leading position Patisiran and giiranvos have taken drug development to a new level, tackling human diseases from an RNA perspective 07
Cenobamate, developed by Cenobamate SK biopharma, was approved by the FDA on November 21, 2019 for the treatment of partial seizures, named Xcopri® epilepsy is a short-lived brain abnormality that can cause uncontrolled activity, abnormal thinking, behavior, and sensation Local epilepsy often begins at restricted sites in the brain According to the CDC, there are about 3 million people with epilepsy in the United States, 60 percent of whom still have seizures after taking AED drugs Cenobamate reduces repetitive neuron discharge by inhibiting the voltage gated sodium flow, and is also a positive regulator for the gaba ion channel However, the specific mechanism of cenobamate for the treatment of epilepsy is not clear Cenobamate's safety and efficacy assessment was based on two randomized, double-blind, placebo-controlled trials that included 655 patients (trial 1, NCT018661111; trial 2, NCT01397968) In trial 1, the median epilepsy frequency in the 200 mg treatment group and the placebo group decreased by 55.6% and 21.5% respectively at 28 days (p 0.0001); The mg group decreased by 36.3% (p-0.006), the 200 mg group decreased by 55.2% (p 0.001), and the 400 mg group decreased by 55.3% (p 0.001) Xcopri ® is the first innovative drug in Korea to receive FDA approval to offer new options for topical epilepsy treatment 08
Voxelotor Voxelotor has been approved by the FDA on 25 November 2019 for the treatment of sickle cell anemia (SCD) in adults and pediatric patients 12 years and older The drug was developed by Global Blood Therapeutics and is called Oxbryta® nearly 100,000 people in the United States are affected by SCD, and millions of people worldwide are affected, especially in Africa SCD is a hereditary blood disease that manifests itself as hemoglobin abnormalities and polymerization, and red blood cells appear like a sickle, reducing the oxygen supply capacity of red blood cells Oxbryta ® is a sickle hemoglobin (HbS) blocker that binds to HbS in accordance with 1:1 to inhibit polymerization by increasing the affinity of hemoglobin to oxygen Oxbryta ® was approved based on a randomized, double-blind, placebo-controlled, multicenter trial (NCT 03036813) 274 patients were treated with Oxbryta ® 1500 mg, 900 mg and placebo, respectively The hemoglobin remission rate was 51.1% in the 1500 mg treatment group, compared with 6.5% in the placebo group (p 0.001) this month, the FDA approved two drugs for SCD Adakveo ® relieves VOC symptoms from a cellular coagulation perspective, while Oxbryta ® relieves protein coagulation from a hemoglobin perspective original title: Global approval of new drugs in November 2019 Overview: 8 in the United States, 2 in China, 1 in the European Union (above)