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Dicerna gets FDA orphan drug status for DCR-PH1 to treat primary hyperoxaluria type 1

Dicerna Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for its therapeutic candidate, DCR-PH1, to treat primary hyperoxaluria type 1 (PH1), a severe, rare, inherited disorder of the liver.

DCR-PH1 is the company’s proprietary DsiRNA-EX-based therapeutic candidate being developed for the treatment of PH1, which often results in kidney failure, and for which there are no approved therapies.

Dicerna product strategy and operations senior vice-president Ted Ashburn said: "The Orphan Drug Designation is an important regulatory milestone as we further our development of DCR-PH1 in PH1, a disease that currently does not have an approved treatment option.

"We are encouraged by the progress of this program to date, and our aim is to rapidly advance the development of DCR-PH1 as a potential new treatment option for PH1 patients."

DCR-PH1 includes a lipid nanoparticle (LNP) technology which allows it to be efficiently delivered to the liver after intravenous (IV) administration.

The company secured rights to this delivery technology under a licensing agreement signed in November 2014 with Tekmira Pharmaceuticals.

DCR-PH1 is designed to address the pathology of PH1 by targeting and destroying the messenger RNA (mRNA) produced by HAO1, a gene implicated in the pathogenesis of PH1.