Recent developments in oligonucleotide therapeutics
This article provides a discussion of the major developments from the last 18 months in the field of oligonucleotide therapeutics. An emphasis is placed on commercial approvals, drugs finishing Phase 3 trials, and clinical trials that were halted due to safety concerns. Market data related to the number of oligonucleotide therapeutics in development are also presented.
The last 18 months have reminded those associated with oligonucleotide therapeutics that drug development is an arduous task filled with dramatic highs and lows.
The beginning of the period included both setbacks and successes leaving many in the field with a sense of optimism tempered with a heavy dose of realism. However, the last few months have brought news of successful Phase 3 clinical trials, more new drug applications (NDAs), and a general feeling that oligonucleotides have finally become an established drug platform. Oligonucleotide therapeutics are synthetically produced DNA or RNA molecules usually 20 to 30 nucleotides in length. These drugs include antisense DNA, RNAi, micro RNA, RNA mimics, aptamers, DNA decoys, immunostimulatory oligonucleotides and oligonucleotide conjugates. The number of oligonucleotide therapeutics approved for commercial sale after more than thirty years of activity in the field stood at 3 in January 2016, and two other drugs not yet approved by the US FDA were being sold in select European countries via Named Patient Supply Studies (1).
2016 was a breakthrough year. Sarepta’s Exondys 51 for the treatment of a type of Duch ...