FDA approves treatment for multiple peripheral neuropathy -- Onpattro (patisiran)

recent, the United StatesThe FDA(http://approved the first treatment for multiple peripheral peripheral neuropathy caused by hATTR,npattro (patisiran)Aboutnpattro (
is the first fda-approvedof siRNAdrug (http://Onpattro is a siRNA therapy that targets thyroxine-carrying proteins (TTR) and restores tissue function by inhibiting the "silence" effect of RNAi on genes by inhibiting the expression of specific mRNAs, removing amyloid protein deposition in tissues and restoring tissue functionthis innovative treatment undoubtedly offers a new treatment option for such patients who come to the United States for medical treatmentthe researchOnpattro's approval is based on data from the Phase III clinical study APOLLOThe study was conducted in a randomized, double-blind, placebo-controlled, global study in patients with hATTR amyloid degeneration with multiple neuropathy to assess the effectiveness and safety of patisiranstudy included 225 patients with hATTR amyloid degeneration with multiple neuropathy, covering 39 genotypes In the study, patients were randomly assigned to patisiran (0.3 mg/kg, intravenously every 3 weeks) and placebo treatment at a ratio of 2:1 　　The results showed that Onpattro achieved excellent therapeutic results, reaching the main and all secondary endpoints of the study: Compared to placebo, Onpattro improved polyneuropathy, quality of life, daily activity, walking ability, nutritional status, and autonomic neurological symptoms In addition, an analysi
s of cardiac subgroup (http:// in patients with myocardial strain (56% of the total number of patients in the study) showed a significant improvement in cardiac structure and functional exploratory endpoints compared to placebo 　　In terms of safety
the incidence and severity of adverse events in the Onpattro treatment group and the placebo group were similar, with higher rates of edema and infusion-related reactions around the Onpattro treatment group, but usually mild to moderate