Shots:

• The US FDA approval is based on P-III ENVISION study assessing Givlaari vs PBO in 94 patients with AHP at 36sites across 18 countries. Givlaari got FDA’s approval in less than four months following its NDA acceptance
• The P-III ENVISION study resulted in 70% fewer porphyria attacks in patients- reduction in intravenous hemin use- reductions in urinary aminolevulinic acid and urinary porphobilinogen. Additionally- in Aug’2019- Alnylam signed a promotional agreement with Ironwood for GIVLAARI to raise awareness of AHP among gastroenterologists and other HCPs in the US
• Givlaari is the world’s first approved GalNAc-conjugate RNA therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) and has received FDA’s BT & ODD and EMA’s PRIME & ODD with its anticipated availability in the US by the year-end

Source 1- Source 2 to­ read full press release/ article | Ref: FDA- Business wire  | Image: Pharmaceutical Technology