英文药名：Refixia（Nonacog Beta Pegol[Genetical Recombination]）
在两个国际协作III期临床试验血友病B患者先前用（FIX活性2％或更少）和目标，年处理在接收本发明的药剂的给药定期患者（40 IU/kg的每周一次）转换出血率如下。 （见表4）
Nonagogue Beta Pegol (Refixia) Hemophilia B therapeutic agent that pegylates factor IX and prolongs its half-life
On July 2, 2018, manufacture and sale of pegylated genetically recombinant blood coagulation factor IX preparation nonacoga beta pegol (commodity name Refixia for IV 500, same 1000 for intravenous injection, 2000 for same) approved It was done. A formulation consisting of a drug vial and a special dissolving solution (L-histidine solution) prefilled syringe. Adaptation is "suppression of bleeding tendency in patients with blood clotting factor IX deficiency", dosage regimen is "dissolved in the total amount of exclusive dissolution solution (4 mL) attached, under various circumstances [at bleeding (mild to moderate, severe or life (Refer to the latest attached document for details) determined for each operation (small bleeding, major surgery), periodic], in any case at a rate not exceeding 4 mL / min Note "has become.
Haemophilia B is a congenital hemorrhagic disease (rare disease) caused by a decrease in plasma FIX activity associated with quantitative or qualitative abnormalities of blood coagulation factor IX (FIX). Specific symptoms are characterized by bleeding which seeps out from deep tissue, most of bleeding sites are subcutaneous bleeding, intra-articular bleeding and intramuscular bleeding. Hemophilia B is mostly a disease seen in men, domestic statistics are affected by 1 to 2 people per 100,000 men and the number of patients is estimated to be about 1000 people.
Treatment is replacement therapy that administers the required amount of FIX formulation reversibly and promptly during bleeding, and regular replacement therapy to prevent progression of haemophilic arthropathy and the like. As a FIX formulation that has been clinically used in the past, it has been proposed to use human plasma derived [dry concentrated human blood coagulation factor IX preparation (Chris machine M, Novact M), dry person blood coagulation factor IX complex formulation (PPSB - HT) There is a preparation of recombinant [blood coagulation Factor IX preparation (Benefit, Lixvis)]. Recently, a FIX agent preparation with extended plasma half-life, gene recombination [blood coagulation Factor IX factor Fc region fusion protein preparation (Orprolix), blood coagulation factor IX albumin fusion protein preparation (IDERBION)] has appeared, The burden on the patient has also been greatly alleviated.
Nonacog Betapagol is a three-component preparation as a FIX agent preparation with prolonged plasma half-life, and is a drug whose half-life is prolonged by modifying (pegylation) the recombinant FIX factor. In the blood clotting process, the activation removes the pegylated activation peptide and converts it into a molecule having the same structure and functional properties as the endogenous active FIX factor, improving the deficiency of the FIX factor, temporal .
In multiple international joint Phase III clinical trials of admission and pediatric hemophilia B patients (including Japanese patients) with treatment history, regular replacement therapy by administration of this drug, hemostatic effect at bleeding, perioperative The effectiveness of the hemostatic effect in the period was confirmed. Overseas, as of May 2018, it is sold in Austria, Canada, Denmark, Germany, the Netherlands, Switzerland and the United States.
In the international joint examination, side effects are recognized in 6.0%. Main side effects are injection site response, pruritus, hypersensitivity, and as a serious side effect, shock, anaphylaxis, thromboembolism may occur, so be careful.
Refixia（Nonacog Beta Pegol 聚乙二醇化重组凝血因子IX注射剂）
简介： 英文药名：Refixia（Nonacog Beta Pegol[Genetical Recombination]） 中文药名：聚乙二醇化重组凝血因子IX注射剂 生产厂家：诺和诺德制药 レフィキシア静注用500／レフィキシア静注用1000／レフィキ ...