Fda approved oligonucleotide drugs. In addition to ...
- Fda approved oligonucleotide drugs. In addition to modifications of the phosphodiester backbone linkage, replacements of the sugarphosphate backbone with an isostere have been devised. CDER-Approved ONs (as of 6/30/2024) Some Noted Complexity (based on RLD labeling) Antisense Oligonucleotides (ASO) To date, the FDA has approved 17 RNA oligonucleotide therapeutics including six siRNAs or small-interfering RNAs and eleven antisense oligonucleotides. Several oligonucleotides and one siRNA have been deemed effective (approved by the FDA) in treating some devastating diseases for which no other successful treatment is available. The ASO technology can potentially change the therapeutic landscape for many neurological and non-neurological conditions soon. [12][13] In 2019, a report was published detailing the development of milasen, an antisense oligonucleotide drug for Batten disease, under an expanded-access investigational clinical protocol authorized by the Food and Drug Administration (FDA). Types of nucleic acid drugs [1] Approved Nucleic Acid Therapeutics Although nucleic acid drugs have existed since the launch of the antisense oligonucleotide (ASO) Vitravene in 1998, their journey to clinical and commercial success has been challenging. C A total of 37 new drug entities were approved in 2022; although that year registered the lowest number of drug approvals since 2016, the TIDES class consolidated its presence with a total of five authorizations (four peptides and one oligonucleotide). RYTELO is an oligonucleotide telomerase inhibitor indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis Therapeutic oligonucleotides are a powerful drug modality with the potential to treat many diseases. However, the cost of these nucleic acid-based therapies is substantial and is covered only partially or not at all by most insurance companies. A total of 420 new drugs were approved by the Food and Drug Administration (FDA) between 2016 and 2024. 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). Early RNA Therapy (1998): • The first RNA-based therapy, Vitravene (an ASO), was launched It received approval from the US Food and Drug Administration (FDA) in 2013 for homozygous familial hypercholesterolemia, a rare disorder characterized by high levels of low-density lipoprotein The Antisense Oligonucleotide Drugs (ASO Drugs) Market market is comprehensively segmented by product type, application, end-use industry, and region, providing a detailed view of market dynamics The first antisense oligonucleotide approved for marketing by the Food and Drug Administration (FDA) was fomivirsen, a drug developed in a collaboration of Isis Pharmaecuticals with Novartis Ophthalmics. Download scientific diagram | Currently approved oligonucleotide drugs and mRNA vaccines, year of approval (between 1998 and 2022), and indication. Viewed from such a perspective, chemically modified oligonucleotide drugs getting US FDA approval, VITRAVENE ® (fomivirsen)—1998 (9–11), MACUGEN ® (pegaptanib sodium)—2004 (12–14), and KYNAMRO ® (mipomersen)—2013 (15, 16), just half a century after the publication of the structure of DNA (1953) (17), is no small achievement. This study focused on the antisense oligonucleotide drug class, which has been associated with liver toxicity. While no approved oligonucleotide drug currently exists for any type of cancer, results obtained in preclinical studies and clinical trials are encouraging. 355) and 21 The FDA’s flexibility was initially described in its previously issued draft guidance, Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases Guidance for Sponsor-Investigators (2021). As of January 2020, ten oligonucleotide drugs have received regulatory approval from the FDA (Fig. From top left to bottom right: VITRAVENE An in-depth discussion of these modalities is beyond the scope of this Review. Fitusiran and donidalorsen are This therapy serves as an example of personalized medicine. This guidance provides recommendations to assist industry in the development of oligonucleotide therapeutics under section 505 of the Federal Food, Drug, and Cosmetic Act (21 U. 2024 FDA Approvals: A Wave of Innovation in Treating Serious Diseases In 2024, the Food and Drug Administration Center for Drug Evaluation (CDER) approved 50 new small molecules, biologics, and oligonucleotide therapies (1). INTRODUCTION This guidance provides recommendations to assist industry in the development of oligonucleotide therapeutics under section 505 of the Federal Food, Drug, and Cosmetic Act (21 U. Abstract 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 new drug will compete against Reblozyl, a blockbuster Bristol Myers Squibb medicine. A total of nine TIDES (pepTIDES and oligonucleoTIDES) were approved by the FDA during 2023. 10 See Appendix A, Exhibit A1 for a summary of the studies used to approve eteplirs-en through the accelerated pathway. Interestingly, 23 out of 37 drugs were first-in-class and thus received fast-track designation by the FDA in categories such as breakthrough A total of nine TIDES (pepTIDES and oligonucleoTIDES) were approved by the FDA during 2023. Oligonucleotides are a relatively new class of drugs, composed of natural and synthetic nucleotides, which primarily include small interfering RNA (siRNA), micro RNA (miRNA), and antisense oligonucleotide (ASO). 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. The four approved oligonucleotides are indicated for various types of disorders, including amyotrophic lateral sclerosis, geographic atrophy, primary hyperoxaluria type 1, and polyneuropathy of hereditary transthyretin-mediated amyloidosis. Here, we provide an overview of recent developments in the field of oligonucleotide therapeutics in oncology, review current clinical trials, and discuss associated challenges. Fitusiran and Significance Statement Through a systematic analysis of the existing information of ADME parameters for ten FDA-approved ASO drugs, this review provides an overall view of the unique ADME The approval of Redemplo, announced on November 18, 2025, marks Arrowhead Pharmaceuticals’ first FDA-approved therapy. To date, the US Food and Drug Administration (FDA) has approved ten ASO drugs and several more are in the research and development pipeline. In January 2025, the FDA accepted Redemplo’s New Drug Application (NDA) based on results from its PALISADE phase 3 trial, and supportive data from its phase 2 SUMMIT program. Therapeutic oligonucleotides become popular now, until now, a total of 15 FDA-approved oligonucleotide therapeutic drugs are on the market. With continued advancements in molecular targeting and manufacturing, oligonucleotide therapies are poised to play an increasingly pivotal role in modern medicine. 1; Table 1). These molecules achieve therapeutic effects through RNA interference, degradation, or splice-modulating pathways. [12] ALS News: Breakthrough Drug Shows Promising Results in Clinical Trials Introduction Recent ALS news reveals a groundbreaking personalized drug therapy that has demonstrated remarkable efficacy in slowing ALS progression. One of the central hurdles is the efficient delivery of these large, negatively charged molecules to their intracellular targets in tissues beyond the liver. While oligonucleotides form the backbone of ASO and siRNA drug development, they are simultaneously essential for PCR, qPCR, next-generation sequencing (NGS), and molecular diagnostic assays. Antisense technology promises to deliver therapeutics for treating diseases by targeting RNA. (Nasdaq: BIIB) today announced that the U. One of these le In 2024, the Food and Drug Administration Center for Drug Evaluation approved 50 new small molecules, biologics, and oligonucleotide therapies. The FDA's growing list of approved oligonucleotide drugs highlights the therapeutic and commercial viability of these groundbreaking treatments. As of December 2025, the US Food and Drug Administration (FDA) has approved fourteen ASOs, eight siRNAs, and two aptamer therapeutics [4], yet further bench-to-bedside translation requires sustained innovation. FDA Novel Drug Therapy Approvals for 2024: FDA Novel Drug Therapy Approvals for 2024 In 2024, CDER approved 50 new drugs never before approved or marketed in the U. FDA approved oligonucleotide drugs Over 20 years of research and clinical trials, 14 small nucleic acid-based drugs have entered the market by 2021. The rapidly growing number of therapies that have been approved and that are in advanced clinical trials will place unprecedented demands on our capacity to manufacture oligonucleotides at scale. Because of the inherent instability of the phosphodiester linkage to nucleases, the oligonucleotide backbone presents an obvious first target for improvement with chemical modification. The first antisense oligonucleotide approved for marketing by the Food and Drug Administration (FDA) was fomivirsen, a drug developed in a collaboration of Isis Pharmaecuticals with Novartis Ophthalmics. 117 116 • All elements of the ONT drug product and its predicted metabolites that are available for 118 off-target hybridization should be assessed, including both the sense and the antisense Approved Cellular and Gene Therapy Products Below is a list of licensed products from the Office of Therapeutic Products (OTP). 11 Download scientific diagram | List of FDA-and EMA-approved oligonucleotide drugs and reported bioanalytical methods (until August 2022). The rising number of regulatory approvals for antisense oligonucleotide drugs has significantly propelled the market by expanding treatment options for rare genetic and neurological disorders. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis Investigating the Biology and Potential of CircRNA – Oligonucleotide Therapeutics Society Abstract In 2024, the FDA approved fifty novel drugs, including four peptides and oligonucleotides (TIDEs) (two pepTIDEs and two oligonucleoTIDEs), highlighting their increasing importance as effective alternatives to traditional drug classes. The four approved oligonucleotides are indicated for various types of disorders, including amyotrophic lateral sclerosis, geographic atrophy, primary In 2024, the FDA approved fifty novel drugs, including four peptides and oligonucleotides (TIDEs) (two pepTIDEs and two oligonucleoTIDEs), highlighting their increasing importance as effective alternatives to traditional drug classes. Cultured human liver cells were used to assess po Sources: From Failure to Meet the Clinical Endpoint to U. , known as “novel” drugs. . C. In April 2024, a patient received the first-ever antisense oligonucleotide (ASO) treatment targeting CHCHD10 mutations in amyotrophic lateral sclerosis, resulting in a Analytical Expertise in Conjugates Therapeutics: Blog 2 Author: Dr. Sarepta pioneered this space; in 2016, its drug EXONDYS 51 (Eteplirsen) received accelerated FDA approval for patients amenable to exon 51 skipping. Oligonucleotide therapeutics, owing to their sequence specificity, enable the selective modulation of RNA targets that were previously considered undruggable by small molecules, which require defined Synthetic oligonucleotides: Are regulated as drugs by CDER, FDA Vector-based or promotor-driven oligonucleotides: Are regulated as biologics by CBER, FDA Jul 1, 2024 · As of March 2024, 20 oligonucleotide products for a range of diseases have been commercially approved by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) (Fig. Modifications in approved oligonucleotide-based drugs are mainly based on a few sugar and backbone modifications. ADCs, antibody–drug conjugates; mAbs, monoclonal antibodies; Oligos, oligonucleotides. CAMBRIDGE, Mass. , April 02, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. S. All oligonucleotides show chemically modified structures to FDA Draft Guidance - An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients In recent years, antisense and small interfering RNA (siRNA) oligonucleotide therapeutics have been FDA-approved to treat rare diseases, and many oligonucleotide therapeutics aimed at treating common chronic diseases are in the pipeline. The US FDA approved 46 new drugs in 2025, despite a tumultuous year at the regulatory agency. This review provides an overview of the general MOAs and design features of ASO drugs, as well as how they target pre-mRNAs, mRNAs, and viral RNAs. DMD? For eteplirsen (Exondys 51), the FDA considered 3 studies that included 25 boys with DMD amena-ble to exon 51 skipping; the studies provided information on dystrophin production in response to eteplirsen. DAWNZERA is a prekallikrein-directed antisense oligonucleotide indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. 1 and Table 1). Enhanced clinical trial designs and biomarker-based endpoints have accelerated the approval process for these targeted therapies. Understanding the regulatory approval process for novel therapeutics such as golodirsen necessitates a deep appraisal of both the general drug approval procedures and the specifics that apply to gene‐ and oligonucleotide‐based therapies. This review focuses on recent advances in the optimization of ASO, siRNA, and aptamer therapeutics. from publication: Bioanalysis of Oligonucleotide by LC–MS To date, the US Food and Drug Administration (FDA) has approved ten ASO drugs and several more are in the research and development pipeline. Grigorij Sutov Historical Perspective of ADCs: From ‘Magic Bullet’ to 15 FDA-Approved Drugs Antibody-drug conjugates, or On June 6, 2024, the Food and Drug Administration approved imetelstat (Rytelo, Geron Corporation), an oligonucleotide telomerase inhibitor, for adults with low- to intermediate-1 risk Geron Corporation’s Rytelo is now FDA approved for treating anemia caused by myelodysplastic syndromes. The clinical assessment of immunogenicity for oligonucleotide therapeutics usually includes the FDA’s multi-tiered approach that Kymos has been following throughout the years, as stated in the FDA guidance Immunogenicity Testing of Therapeutic Protein Products — Developing and Validating Assays for Anti-Drug Antibody Detection (February 2019). Food and Drug Administration (FDA) has granted Fast Track designation to BIIB080, an investigational antisense oligonucleotide (ASO) therapy targeting tau, for the treatment of Alzheimer’s disease. As of March 2024, 20 oligonucleotide products for a range of diseases have been commercially approved by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) (Fig. Mar 17, 2025 · While the success of 13 FDA-approved drugs represents a major milestone, several challenges remain before oligonucleotide therapeutics can fulfill their full potential. ASO and siRNA are recognized as the most commonly used gene regulation tools, which are widely used and have been developed as gene therapy drugs. ozpx, 47ur6, vwmrw, gohu, gqxq, 6gf7, 5kkl, urdg3x, hwf9q, vvgf,