A recent press release by the company Regulus Therapeutics highlights their newest orphan drug RG-012 – a microRNA treatment designed to help patients with Alport Syndrome, a life-threatening genetic kidney disease that impacts the body’s ability to create a specific type of collagen highly expressed in the kidney and essential to normal kidney structure. The release neglects to mention is that this is also one of the first cases of the FDA applying Orphan Drug designation to a microRNA treatment.
A relatively new discovery, these small oligonucleotides do not code for proteins but instead regulate gene expression, often regulating entire pathways. Being able to modulate the expression of miRNA has the potential to impact many diseases, and Regulus’s research in the field is paving the way for future FDA approvals. Amy Conrad, the Senior Director of Investor Relations at Regulus, mentioned that Regulus hopes to see the FDA begin to more readily understand the technologies and potential applications surrounding the miRNA.
This would ultimately lead to more (and less complicated) drug approvals down the road. Like any new technology, it often takes time for regulation to catch up to reality. What they call “anti-sense therapy” is bound to grow in the coming years, and the importance of this designation is the exposure the clinical trial process of this drug will bring to the fledgling technology.
To learn more about Orphan Drugs, see the FDA website here: Orphan Drugs. If you are interested in companies like Regulus pursuing Orphan Drug designation, be sure to stop by World Orphan Drug Congress 2015 taking place April 23-24 in Washington, D.C. [Image: DNA/Naturalis Leiden– Flickr]