In 2025, the FDA approved 46 novel drugs, including four TIDEs (one peptide and three oligonucleotides), expanding treatments for rare diseases such as Barth syndrome and hereditary angioedema.
The 2025 FDA approvals of novel peptide and oligonucleotide therapeutics underscore the continued advancement and clinical maturity of these drug classes.
In 2025, the FDA approved 46 novel drugs, including four TIDEs (one peptide, three oligonucleotides, and one antibody drug conjugate containing peptide as a payload). The three approved oligonucleotide therapeutics—fitusiran, donidalorsen, and plozasiran—bring the total number of approved oligonucleotide drugs to 24 across 16 clinical indications since 1998. Fitusiran and donidalorsen are the first oligonucleotide therapies approved for antithrombin deficiency and hereditary angioedema, respectively, while plozasiran represents the second approved therapy for familial chylomicronemia syndrome. All three agents employ GalNAc-mediated hepatocyte targeting, highlighting the continued importance of liver-directed delivery platforms in oligonucleotide drug development and underscoring the growing clinical maturity of this therapeutic class. Peptide-based therapeutics continue to emerge as pioneering treatments for longstanding diseases. In 2025, elamipretide further expanded this paradigm by becoming the first disease-specific treatment approved for Barth syndrome. This review provides an overview of TIDES approved in 2025, with emphasis on their chemical structures, medical targets, modes of action, routes of administration, and associated adverse effects.
AlShaer et al. (2026) conducted a review in Antithrombin deficiency, hereditary angioedema, familial chylomicronemia syndrome, Barth syndrome. TIDEs (Peptides and Oligonucleotides) was evaluated. In 2025, the FDA approved 46 novel drugs, including four TIDEs (one peptide and three oligonucleotides), expanding treatments for rare diseases such as Barth syndrome and hereditary angioedema.