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Breakthrough Drug Introduced to Treat Primary Hyperoxaluria Type 1

Dicerna Pharmaceuticals announced that its investigational drug, DCR-PHXC is used for the treatment of patients with primary hyperoxaluria type 1 (PH1), a rare genetic liver disorder that often results in kidney failure.

It has received a Breakthrough Therapy Designation (BTD) from the US Food and Drug Administration (FDA).

The FDA also concluded that PH type 2 (PH2) and PH type 3 (PH3) meet its criteria for a serious or life-threatening disease or condition. The biopharmaceutical company said that it will maintain a dialogue with the FDA regarding endpoints for studies of DCR-PHXC in patients with PH2 and PH3, as part of the PHYOX clinical development program.

Recently, Dicerna demonstrated significant post-dose reductions in 24-hour urinary oxalate levels in adults and adolescent patients with PH1 and PH2. Additionally, it also showed that the urinary oxalate levels in a majority of patients were normalized or near-normal after a single dose of DCR-PHXC and was generally well-tolerated.

The drug DCR-PHXC in animal models of PH silences lactate dehydrogenase A enzyme, or LDHA, in the liver. As a result, there will be a blockage of the excess production of oxalate. In preclinical studies of DCR-PHXC, the compound was well-tolerated with no adverse effects in the liver.

As per study, it showed that people who are completely deficient in LDHA showed no liver dysfunction and can lead normal lives. For patients with PH, the LDHA deficiency in the liver may be beneficial as the LDHA enzyme is implicated in the abnormal production of oxalate in PH. This in turn is responsible for the severe damage to other organs and kidneys in patients with PH.

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