FDA approves first PH1 medicine that slows progression of kidney disease

For people who have been living with the rare genetic disease primary hyperoxaluria type 1 (PH1), encouraging news is on the horizon. The most recent U.S. Food and Drug Administration (FDA) treatment approval of Oxlumo™ (lumasiran) by Alnylam Pharmaceuticals, Inc. is the first therapy for people who have been diagnosed with PH1 and are awaiting a treatment option.

PH is a rare genetic disease that begins in the liver and causes a waste product called oxalate to build up in the body. Oxalate is normally filtered through the kidneys and out of the body through the urine (pee). In people with PH1, the buildup of oxalate is deposited in the kidneys and urinary tract. PH1 can lead to frequent kidney stones, progression of kidney disease and systemic organ dysfunction. If left untreated, PH1 can lead to kidney failure, requiring dialysis or a kidney transplant to stay alive.

As a rare genetic disease, PH1 is a challenging condition to diagnose and treat. Even among members of the same family, the age of diagnosis and rate of disease progression can vary significantly for people with PH1. Until Oxlumo™, liver transplantation was the only preemptive treatment to address the underlying cause of oxalate overproduction in people with PH1.

The FDA approval follFor people who have been living with the rare genetic disease primary hyperoxaluria type 1 (PH1), encouraging news is on the horizon. The most recent U.S. Food and Drug Administration (FDA) treatment approval of Oxlumo™ (lumasiran) by Alnylam Pharmaceuticals, Inc. is the first therapy for people who have been diagnosed with PH1 and are awaiting a treatment option.

PH is a rare genetic disease that begins in the liver and causes a waste product called oxalate to build up in the body. Oxalate is normally filtered through the kidneys and out of the body through the urine (pee). In people with PH1, the buildup of oxalate is deposited in the kidneys and urinary tract. PH1 can lead to frequent kidney stones, progression of kidney disease and systemic organ dysfunction. If left untreated, PH1 can lead to kidney failure, requiring dialysis or a kidney transplant to stay alive.

As a rare genetic disease, PH1 is a challenging condition to diagnose and treat. Even among members of the same family, the age of diagnosis and rate of disease progression can vary significantly for people with PH1. Until Oxlumo™, liver transplantation was the only preemptive treatment to address the underlying cause of oxalate overproduction in people with PH1.

The FDA approval follows positive results of the ILLUMINATE clinical study to demonstrate the safety and efficacy of Oxlumo™ among adults and children. In the study, adult patients taking Oxlumo™ had a 65% decrease in oxalate and children patients had a 72% decrease in oxalate. The ILLUMINATE study is the largest study specifically for PH1, conducted in eight countries around the world.

Clinical trials were a critical part of developing this new medicine. To learn more about other clinical trials for kidney patients, please visit our clinical trials resource page. For more information about primary hyperoxaluria and PH1, visit KidneyFund.org/ph.ows positive results of the ILLUMINATE clinical study to demonstrate the safety and efficacy of Oxlumo™ among adults and children. In the study, adult patients taking Oxlumo™ had a 65% decrease in oxalate and children patients had a 72% decrease in oxalate. The ILLUMINATE study is the largest study specifically for PH1, conducted in eight countries around the world.

Clinical trials were a critical part of developing this new medicine. To learn more about other clinical trials for kidney patients, please visit our clinical trials resource page. For more information about primary hyperoxaluria and PH1, visit KidneyFund.org/ph.