Alnylam Reports Results of Givosiran in P-III ENVISION Study to Treat Acute Hepatic Porphyria

Shots:

• The P-III ENVISION study involves assessing of Givosiran vs PBO in patients with acute hepatic porphyria (AHP)
• ENVISION study results:74% mean reduction of annualized rate of porphyria attacks; 90% median reduction in composite annualized attack rate (AAR); overall safety and tolerability; AEs (89.6% vs 80.4%); attack free (50% vs 16.3%); @3/6mos. 91%/83% in urinary ALA; @6mos. 73% in urinary levels of PBG; 77% in no. of annualized days on hemin; 73% in composite AAR for patients with any AHP   
• Givosiran is an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) with its expected NDA and MAA submission in mid-2019

Click here to read full press release/ article | Ref: Alnylam | Image: Investor’s Business Daily