Innovative RNAi therapy identified by FDA breakthrough therapy for the treatment of hereditary liver disease

PH is a serious and rare genetic liver disease in which excessive oxalate is produced in the patient's body, which accumulates in the kidneys and cannot be discharged in timePatients with decreased renal function can develop oxalate deposition, which can lead to the accumulation of oxalateins in bones, skin, heart, retina and other areas, and serious damage to the kidneys and related organsPatients with severe PH often require a liver or kidney transplant and are given immunosuppressive drugs for lifeThree types of PH are known to exist, PH1 is caused by mutations in the AGXT gene, resulting in a lack of alanine-acetaldehyde amino transferase (AGT)There are more than 5,000 PH1 patients in the United States and the European Union, but there is currently no approval to treat the diseaseDCR-PHXC is the only RNAi drug currently used to treat all types of primary hyperoxalic acid (PH), using Dicerna's original GalXC technology platform to selectively silence lactic acid dehydrogenase A (LDHA) in the liver to block the production of excess oxalateThe drug is well tolerated in preclinical studies and has no adverse effects on the liverDCR-PHXC mechanism (Photo Source: References)in the Phase 1 clinical trial of DCR-PHXC therapeutic PH, the level of oxalate in the urine decreased significantly 24 hours after administration in patients with PH1 and PH2Single-dose DCR-PHXC treatment allows oxalate levels in most patients to reach normal or near-normal in their urine and have good toleranceat the same time, the FDA also confirmed that PH2 and PH3 meet the criteria for serious or life-threatening diseasesDicerna also communicates with the FDA on an ongoing basis about the end of DCR-PHXC's treatment of PH2 and PH3"The FDA recognizes the urgency of developing treatments for type 1 primary hyperoxyuria, and DCR-PHXC's Phase 1 clinical trial has yielded positive results and has been awarded breakthrough therapy," said DrRalf Rosskamp, Chief Medical Officer of Dicerna"
Reference sRetrieved July 16, 2019, from Retrieved July 16, 2019, from Retrieved July 16, 2019, from