Shots:

• The P-III ENVISION study involves assessing of Givosiran vs PBO in 94 patients with AHP including patients with acute intermittent porphyria (AIP) across 18 countries in ratio 1:1             
• The ENVISION study resulted in meeting 1EPs & 2EPs i.e- reduction in porphyria attacks- presented at EASL on 13 Apr-2019 with its expected NDA & MAA filing in H2’19
• Givosiran (SC- 2.5mg/kg/mo) is a RNAi therapeutics- based on Alnylam’s Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology. It has received FDA’s BT & EMA’s PRIME designation and ODD in both- the US & EU

Ref: Alnylam | Image: FDA Headlines