News Feature | July 23, 2014

Regulus Receives FDA Orphan Status For Rare Kidney Disease Drug

By Estel Grace Masangkay


Biopharmaceutical company Regulus Therapeutics announced that the U.S. Food and Drug Administration (FDA) have issued Orphan Drug designation to its RG-012 for the treatment of rare kidney disease Alport syndrome.

RG-012 is being developed as a single stranded, chemically modified oligonucleotide designed to bind to and inhibit the function of miR-21. miR-21 is observed to be overexpressed in mouse models of the rare disease. Inhibition of miR-21 resulted in an increase of the mice’s lifespan by up to 50 percent and decrease in the progression rate of renal fibrosis.

Alport syndrome is a life threatening genetic kidney disease that hampers patients’ ability to produce a certain collagen needed for normal kidney structure. Alport syndrome patients’ kidneys cannot effectively filter toxins and waste leading to end-stage renal disease. Hearing loss and affected vision are also reported as symptoms. There is currently no approved treatment for Alport syndrome.

Paul Grint, CMO of Regulus, said, “We are pleased to have received orphan drug designation for RG-012 and are encouraged by the FDA's recognition for the need of innovative new treatments like microRNA therapeutics for rare and orphan diseases such as Alport syndrome… We believe that RG-012 represents an opportunity to make a significant impact in the lives of patients with Alport syndrome and we look forward to advancing this program into the clinic.” Regulus recently appointed Mr. Grint last month to its executive management team.

The company said it expects to start a Phase I clinical study of RG-012 in the first half of 2015.

Last month Regulus announced that it has received patent grant for its microRNA therapeutics for the treatment of hepatitis C virus from the U.S. Patent Office in Sarnow Patent Estate. The patent grant includes the use of a wide class of anti-miR inhibitors of MiR-122 for the treatment of HCV as a single agent or in combination with other anti-viral drugs.