Shots:

• The P-III HELIOS-A study assessing Vutrisiran (25mg- SC- once every 3mos.) vs patisiran (0.3 mg/kg- IV- q3w) in 164 patients in a ratio (3:1) with hATTR amyloidosis with polyneuropathy
• The study met its 1EPs & 2EPs i.e. change from baseline in mNIS+7 @9mos. & improvement in QoL assessed by Norfolk QoL-DN and gait speed assessed by timed 10-MWT. The therapy showed improvements in exploratory EPs @9mos.- including NT-proBNP- overall disability and measures nutritional status
• The company has submitted an NDA to FDA and plans to file the regulatory filing in Brazil and Japan and MAA in the EU upon obtaining the results of the 18mos. analysis from the P-III HELIOS-A study

  Ref: Business Wire | Image: Stat News