Shots:

• The P-III ILLUMINATE-B study involves assessing Lumasiran in 18 patients with PH1 under the age of 6 (3-72mos.)- with an eGFR >45 mL/min/1.73 m2 or normal serum creatinine if less than 12mos. old at 9 study sites in 5 countries across the globe
• The study demonstrated a reduction in the hepatic production of oxalate across all ages- safety & tolerability profile is consistent with that observed in P-III ILLUMINATE-A study. The study also demonstrated positive results across 2EPs- including additional measures of urinary and plasma oxalate
• Lumasiran (SC) RNAi therapeutic targeting HAO1 in development for the treatment of PH1 and has received the US FDA’s ODD & BT in addition to EMA’s ODD and PRIME designation

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