Rare Inherited Liver Disorder Therapy Granted Orphan Drug Status
Dicerna Pharmaceuticals announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to DCR-PH1 for the treatment of primary hyperoxaluria type 1 (PH1).
In preclinical studies, DCR-PH1 has been shown to induce potent and long-term inhibition of HAO1 and to significantly reduce levels of urinary oxalate, while demonstrating long-term efficacy and tolerability in animal models of PH1.
DCR-PH1 is engineered to target and destroying the messenger RNA (mRNA) produced by HAO1, a gene implicated in the pathogenesis of PH1. HAO1 encodes glycolate oxidase, a protein involved in producing oxalate. Currently there no approved therapies for this severe, rare inherited liver disorder.
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