FDA-Approved Oligonucleotide Therapies in 2017

Area of Science:

• Nucleic acid therapeutics
• Oligonucleotide drug development
• Clinical pharmacology

Background:

• Oligonucleotides, including antisense oligonucleotides (ASOs), aptamers, and small interfering RNAs (siRNAs), have been in clinical development for decades.
• Thousands of participants have been enrolled in numerous clinical trials evaluating these molecules.
• Regulatory assessment identifies specific oligonucleotide therapeutics with proven clinical benefit.

Purpose of the Study:

• To review the development and clinical success of oligonucleotide therapeutics.
• To highlight the six oligonucleotide drugs assessed by regulatory authorities as of January 2017 for providing clear clinical benefit.
• To provide a historical perspective on oligonucleotide-based therapies.

Main Methods:

• Review of clinical trial data and regulatory assessments of oligonucleotide therapeutics.
• Analysis of molecules that have reached advanced stages of clinical development.
• Identification of drugs demonstrating clear clinical benefit in controlled trials.

Main Results:

• Six oligonucleotide therapeutics were identified as providing clear clinical benefit based on rigorous trials as of January 2017.
• The review details the clinical journey of these successful oligonucleotide drugs.
• Significant investment in oligonucleotide research has led to approved therapies.

Conclusions:

• Oligonucleotide therapeutics have evolved significantly over 30 years of clinical development.
• A select group of oligonucleotide drugs has proven effective and beneficial in clinical settings.
• Continued research and development in oligonucleotide technology holds promise for future therapies.