ALNY - Alnylam gets FDA nod for expanded use of rare disease drug Oxlumo

• The U.S. Food and Drug Administration (FDA) approved the expanded use of Alnylam Pharmaceuticals ( NASDAQ: ALNY ) Oxlumo (lumasiran) subcutaneous injection for treating primary hyperoxaluria type 1 (PH1) to lower urinary oxalate (UOx) and plasma oxalate (POx) levels in pediatric and adult patients.
• The company's supplemental new drug application ((sNDA)) was backed by data from a phase 3 trial called ILLUMINATE-C which evaluated Oxlumo in patients with severe renal impairment, including those on hemodialysis.
• The sNDA also included data from open-label extensions of Phase 3 studies, ILLUMINATE-A and ILLUMINATE-B, in pediatric and adult patients with PH1.
• PH1 is a ultra-rare genetic disease characterized by oxalate overproduction in the liver. High levels of oxalate crystals in the kidneys and urinary tract and can lead to formation of kidney stones, progression to kidney failure, and even systemic organ dysfunction, the company said in an Oct. 6 press release.
• The company noted that in November 2020, Oxlumo was approved by the FDA to treat PH1 to lower UOx levels in pediatric and adult patients.

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