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news.US-based biopharmaceutical firm Regulus Therapeutics has received orphan drug designation from the US Food and Drug Administration (FDA) for RG-012, a single stranded, chemically modified oligonucleotide.
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RG-012 binds to and inhibits the function of microRNA-21 (miR-21), as a therapeutic to treat Alport syndrome, a genetic kidney disease.
In the near term, the company intends to start a natural history of disease trial to gather further information about the progression of Alport syndrome and to inform future clinical development plans of RG-012, a key program under its ‘Clinical Map Initiative’.
Regulus chief medical officer Paul Grint said the company is happy to secure orphan drug designation for RG-012 and are encouraged by the FDA’s recognition for the need of new treatments like microRNA therapeutics for rare and orphan diseases, such as Alport syndrome.
"Alport syndrome is a life threatening disease and patients have very limited treatment options because there is currently no approved therapy," Grint said.
"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."
The company had discovered that miR-21 is highly overexpressed in mouse models of Alport syndrome.
According to the company, miR-21 has showed potent inhibition of miR-21 in vitro and in vivo, a decrease in the rate of progression of renal fibrosis, an increase in the lifespan of mice by up to 50%, and a favorable pharmacokinetic profile that supports the potential for a once per week dosing regimen.