FDA approves Onpattro infusion therapy for adult patients with peripheral neurological disorders

recently, the U.SFDA(announced the approval of Onpattro (patisiran) infusion therapy for adult patients with peripheral neuropathy (polyneuropathy) caused by hereditary thyroxine amyloid degeneration (hATTR)on OnpattroDeveloped by Alnylam, Onpattro is a siRNA therapy that targets transthyretin (TTR), adrug that(through a silent part of the RNA involved in the disease)Onpattro is the form of siRNA wrapped in lipid nanoparticles that deliver drugs directly to the liver in infusion therapy, interfering with the production of RNA in the abnormal form of TTRBy preventing the production of TTR, Onpattro can help reduce the accumulation of amyloid deposits in the peripheral nerves, improve symptoms, and help patients better control the conditionOnpattro has been awarded by the U.SFDA for breakthrough therapy, priority review eligibility, fast-track eligibility, and orphan drug eligibilitystudiesonpattro's efficacy was also demonstrated in clinicaltrialIn a study of 225 patients, 148 patients were randomly assigned to receive Onpattro infusions every three weeks for a total of 18 months, and 77 patients were randomly assigned to receive the same frequency of placebo infusionsresults showed that patients treated with Onpattro had better results in the measurement of multiple neuropathy, including muscle strength, sensation (pain, body temperature, numbness), reflexes, and autonomic nerve symptoms (blood pressure, heart rate, digestion) than those who received a placebo infusionPatients treated with Onpattro also scored higher on the assessment of walking, nutritional status, and ability to perform daily activities