Treatment of hemophilia among young patients Sanofi innovative RNAi therapy approved clinically in China

On August 13, The National Drug Administration's Drug Review Center (CDE) announced that Sanofi's Genzyme company had obtained an implicit license for a clinical trial of the new drug fitusiran injection, which is used to treat adult patients with or without inhibitory antibodies in adult patients with type A or Type B haemophilia and adolescents aged 12 and over (including 12 years of age) to prevent or reduce the frequency of bleedingscreenshot source: CDE's official websiteThis is a new RNAi treatment developed by Genzyme in partnership with AlnylamThe latter may be no stranger to the company's first RNAi therapy, Patisiran, which was approved by the U.SFDA in August 2018Fitusiran is the company's most anticipated RNAi treatmenthaemophilia is a genetic bleeding disorder characterized by the failure to produce the level of coagulation required for effective clotting, which leads to recurrent bleeding in joints, muscles and major internal organsPublic data show that about 200,000 people worldwide are diagnosed with haemophilia A or BCurrently, standard treatment for haemophiliacs includes alternative therapy for coagulation factors, which can temporarily restore the body's clotting capacity preventively or "on-demand"However, more than 1/3 of severe haemophiliacs of type A will neutralise their replacement factors, which in turn can lead to very serious complicationsAs a result, such patients are in urgent need of drug options other than standard alternative factorsFitusiran is a monthly subcutaneous injection of RNAi anticoagulase (antithrombin (AT) therapy designed to lower AT levels and promote the production of enough clotting enzymes to restore bleeding and prevent bleedingAlnylam developed the therapy using its ESC-GalNAc conjugate technology, which has increased the effectiveness and durability of subcutaneous administrationIn addition, there is potential to treat other rare haemorrhagic diseasesIt is reported that as a strategic partner, Sanofi has invested significant funds to support the development of the therapypreviously, the clinical results of Phase 1 of the therapy showed initial safety and toleranceStudies have shown that fitusiran can reduce AT levels and increase the production of thrombin in patients with type A and B haemophilia without inhibitors Subsequent phase 2 studies showed that fitusiran's safety and tolerability data were equally encouraging during the co-administration of alternative factors or bypass drugs, and that there were no thrombosis incidents According to the latest data from ClinicalTrials.gov, there are currently five clinical trials of the therapy in progress, and we expect these trials to yield positive results : ClinicalTrials.gov
The pattern of hemophilia treatment in China has changed a lot in the past two years, and more and more innovative therapies have come to patients at a faster pace as regulators continue to push forward a series of reforms We congratulate this innovative hemophilia RNAi treatment on its approval of clinical treatment in China and look forward to further promoting clinical research for the early benefit of more haemophiliacs original title: Courier - Sanofi Innovative RNAi Therapy Approved clinically in China to Treat Hemophilia