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    Home > Medical News > Medical World News > Overview of new drug approval in November: 8 in the United States, 2 in China and 1 in the European Union (I)

    Overview of new drug approval in November: 8 in the United States, 2 in China and 1 in the European Union (I)

    • Last Update: 2019-12-09
    • Source: Internet
    • Author: User
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    In November 2019, the world's first approval of new drugs mainly concentrated in the United States, China and the European Union Us food and Drug Administration (FDA) approved six new molecular substance (NME) as contrast agent air polymer type for the detection of fallopian tube patency A Cefiderocol for the treatment of complex urinary tract infection, zanubrutinib for the treatment of mantle cell lymphoma, givosiran for the treatment of acute intermittent porphyria, cenobamate for the treatment of partial seizure epilepsy and voxelotor for the treatment of sickle cell anemia The National Drug Administration (nmpa) approved two NME drugs, namely, the sodium oligoannurate for Alzheimer's disease and flumatinib for chronic myeloid leukemia In addition, FDA approved two new biological products, namely, lustatercept for the treatment of thalassemia and crizanlizumab for the treatment of sickle cell anemia The European Drug Administration (EMA) approved a new vaccine for the prevention of rbsv EBOV infection Airport type A has been approved by FDA on November 7, 2019 for sonohysterosalpingography to assess tubal patency in women with known or suspected infertility The drug was developed by giskit B.V under the trade name of execm ® [1] About 12-33% of infertility cases are caused by fallopian tube obstruction Therefore, the evaluation of fallopian tube patency is a routine item to detect infertility [2,3] Exem ® consists of hydroxyethyl cellulose, an echo contrast agent When visualized by ultrasound, the foam will display an echo or bright signal [4] in the fallopian tube and peritoneal cavity The approval of ExEm is based on two studies of uterine oviduct ultrasound bubble imaging (HyFoSy) In test a, 80% of the tubal obstruction and 92% of the tubal patency were assessed by 2D imaging, and 98% and 91% by 3D imaging respectively Test B combined with 2D / 3D imaging technology, the detection results are similar to test a [4] Luspatercept AAMT has been approved by FDA on November 8, 2019 for the treatment of β - thalassemia requiring regular red blood cell transplantation, under the trade name of reblazyl ® The drug was initially developed by accelerator and then authorized to new base company [5] Normal human hemoglobin is composed of two α - globins and two β - globins β - thalassemia is due to the defect or mutation of the gene encoding β - globin, the decrease of β - globin synthesis, and the excess of α - globin, which induces the apoptosis of erythrocytes in the form of inclusion body The incidence rate of the disease is 1/100000 worldwide, while the European Union is 1/10000 Cyprus (incidence rate 14%), Sardinia (10.3%), Southeast Asia and other countries and regions are the most serious [6] At present, long-term blood transfusion combined with iron chelating agent and hematopoietic stem cell transplantation are the main treatment options, but there are shortcomings such as iron deposition and HLA matching difficulties Reblozyl ® is a recombinant fusion protein targeting TGF - β family ligands, which can reduce the effect of Smad2 / 3 signaling pathway The drug can promote the differentiation of late precursor erythrocytes and enhance the proliferation of erythrocytes [7] Reblozyl ® was approved in a multicenter, randomized, double-blind, placebo-controlled trial (nct02604433) 336 patients with thalassemia were enrolled and received reblozyl ® or placebo 2:1 21% of the patients in the treatment group had at least 33% reduction in blood transfusion volume, while only 4.5% of the patients in the placebo group achieved this index (P < 0.0001) [7] Reblozyl ® is the first approved erythropoietic maturation drug in the United States, which can relieve patients' dependence on blood transfusion Cefiderocol, developed by yanye pharmaceutical and approved by FDA on November 14, 2019, is used for the treatment of complicated urinary tract infection (CTU) Its trade name is fetroja ® In addition, the drug also submitted an application for listing to the EU EMA in April 2019 [8] The common pathogenic bacteria of CTU include Gram-negative bacteria such as Escherichia coli, Klebsiella, Pseudomonas, and some gram-positive bacteria such as Enterococcus, which mainly show the structural and functional abnormalities of the urogenital system [9] In clinic, it is often treated with broad-spectrum antibiotics, operation and catheterization [10] Cefiderocol is a cephalosporin antibiotic for Gram-negative bacteria, and it can be used as an iron carrier to absorb the free iron Cefiderocol can enter the periplasmic space of bacteria through the passive diffusion of pore proteins or through the active transport mechanism of iron carriers Cefiderocol can inhibit the biosynthesis of bacterial cell wall by binding with penicillin binding protein family (PBPs) [11] Cefiderocol was approved based on an international multicenter, randomized, double-blind trial (nct02321800) in which 448 inpatients of CTU were treated with cefiderocol or imipenem / cilastatin 2:1 The results showed that 72.6% of the patients in cefiderocol group achieved bacteriological clearance and clinical remission on the 7th day of treatment, compared with 54.6% in the control group [11] The high mortality rate caused by CTU caused by gram-negative bacteria is a medical challenge, and yanye believes that fetroja ® will become one of the best drug treatment options Zebutini, developed by Baiji Shenzhou, has been approved by the FDA for marketing on November 14, 2019, and is used for the treatment of adult mantle cell lymphoma (MCL) Its trade name is brukinsatm [12] MCL is a B-cell non-Hodgkin's lymphoma, the main incidence of the population for the elderly MCL starts from lymph nodes and gradually spreads to spleen, bone marrow, blood, even esophagus, gastrointestinal and other parts According to the American Cancer Association, MCL accounts for about 5% of lymphoma [13] Brukinsatm is a small molecule Btk (Bruton tyrosine kinase) inhibitor, which can block the related signal transmission, thus inhibiting the growth of malignant proliferation B cells and killing tumor cells [14] Brukinsatm was approved based on a clinical phase II, open label, multicenter, single arm trial (nct03206970) involving 86 previously treated MCL patients The results showed that 84% of the patients achieved tumor mass reduction, and the median total remission period was 19.5 months Another single arm trial (nct02343120) included 32 patients, 84% of whom had shrunk tumor mass, with a median total remission period of 18.5 months [14] Brucinsatm not only breaks through the side effects of ibuprofen on other targets, but also greatly improves the drug tolerance concentration Since 2015, China has carried out the reform of drug administration, which has increased the enthusiasm of a large number of domestic pharmaceutical enterprises in R & D Based on the favorable domestic policy environment, brukinsatm has become the first Chinese original drug to go overseas Crizanlizumab has been approved by FDA on November 15, 2019 The drug was developed by selexys Pharma (acquired by Novartis in 2012) for the treatment of vascular occlusive crisis (VOCs) in sickle cell disease patients The trade name is adakveo ® [15] Sickle cell anemia is a kind of complicated and failing blood genetic disease, which involves not only red blood cells The clinical manifestations are chronic inflammation, the increase of P-selectin and other cell adhesion proteins, cell aggregation and blood flow blockage, which lead to vascular occlusive crisis (VOCs) and other life-threatening complications VOCs are the main inducement for sickle cell anemia patients to be hospitalized There are about 200000 Er emergency cases in the United States every year [16] Adakveo ® is a humanized IgG2 kappa monoclonal antibody targeting P-selectin, which can block the interaction between P-selectin and its ligand, such as PSGL-1 Adakveo ® inhibits the aggregation of endothelial cells, red cells and white cells by binding to P-selectin on activated endothelial cells and platelets [17] Adakveo ® was approved based on a sustain clinical study (nct01895361) The median annual incidence of VOC in the treatment group was lower than that in the placebo group (1.63 vs 2.98, P = 0.010) About 36% of patients in the treatment group had VOC events, compared with 17% in the placebo group The median time for the first occurrence of VOC in the treatment group and placebo group was 4.1 months and 1.4 months respectively [17] Adakveo ® is the first McAb targeting P-selectin, which is of great significance for drug research and development in the field of SCD Givosiran, developed by Alnylam, was approved by FDA on November 20, 2019 for the treatment of adult acute porphyria (AHP) under the trade name givlaari ® [18] AHP is an extremely rare genetic disease, which endangers the life and quality of life of patients There are four types of AHP: acute intermittent porphyria (AIP), hereditary fecal porphyria (HCP), variable porphyria (VP) and autosomal recessive 5-aminolevulinate dehydratase deficient porphyria (ADP) The overall incidence rate of AHP was 5/10 million, while the genetic exportability of AIP, HCP and VP was very low About 90% of the heterozygotes carrying pathogenic mutations could survive asymptomatic [19] Givlaari ® is a small interfering RNA directed by 5-aminolevulinate-1 (ALAS1), which can reduce the elevated ALAS1 mRNA level in hepatocytes by RNA interference [20] Givlaari ® was approved based on a randomized, double-blind, placebo-controlled international trial (nct03338816) 94 patients were enrolled into the treatment group or placebo group according to 1:1 Compared with placebo, the givlaari ® group had a 70% reduction in porphyrin attack [20] RNAi technology has been rated as the top ten scientific and technological progress in 2001 by science magazine In August 2018, Alnylam launched paisiran, the world's first RNAi drug, while the approval of givesiran and other drugs under research showed Alnylam's leading position Paisiran and givosiran have pushed drug research and development to a new level, attacking human diseases from the perspective of RNA Cenobamate, developed by SK biopharma, has been approved by FDA on November 21, 2019 for the treatment of partial seizure epilepsy The trade name is xcopri ® [21] Epilepsy is a transient abnormal brain potential activity, which can cause uncontrolled activities, abnormal thinking, behavior and feeling Local epilepsy often starts at the brain's restricted site According to the CDC statistics of the United States, there are about 3 million epileptics in the United States, 60% of them still have seizures after taking AED drugs [22] Cenobamate can reduce repetitive neuronal discharge by inhibiting voltage-gated sodium influx, and it is also a positive allosteric regulator of GABA ion channel However, the specific mechanism of cenobamate in the treatment of epilepsy is not clear [23] The safety and efficacy evaluation of cenobamate was based on two randomized, double-blind, placebo-controlled trials involving 655 patients (Trial 1, nct01866111; trial 2, nct01397968) In trial 1, the median frequency of epilepsy decreased by 55.6% and 21.5% (P < 0.0001) at 28 days in the 200 mg treatment group and placebo group respectively; in trial 2, the median frequency of epilepsy decreased by 24.3% in the placebo group, 36.3% (P = 0.006) in the 100 mg group, 55.2% (P < 0.001) in the 200 mg group and 55.3% (P < 0.001) in the 400 mg group [23] Xcopi ® is the first FDA approved innovative drug in South Korea, providing a new choice for the treatment of local epilepsy Voxelotor has been approved by FDA on November 25, 2019 for the treatment of sickle cell anaemia (SCD) in adults and pediatric patients aged 12 and over The drug was developed by global blood therapeutics under the trade name of oxbryta ® [24] Nearly 100000 people in the United States are affected by SCD, and millions of people in the world are affected, especially in Africa [25] SCD is a kind of hereditary blood disease, which is characterized by abnormal and aggregation of hemoglobin, sickle shape of red blood cells, and reduction of oxygen supply capacity of red blood cells [26] Oxbryta ® is a sickle hemoglobin (HBS) inhibitor, which binds to HBS in 1:1 ratio, and inhibits polymerization by increasing the affinity of hemoglobin to oxygen [27].
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