Eteplirsen
- Generic Name
- Eteplirsen
- Brand Names
- Exondys
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1173755-55-9
- Unique Ingredient Identifier
- AIW6036FAS
- Background
Eteplirsen is a synthetic antisense oligonucleotide and a phosphorodiamidate morpholino oligomer. It consists of a six-membered morpholino ring replacing the five-membered ribofuranosyl rings found in natural DNA and RNA.
Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle deterioration and premature death most commonly due to respiratory or cardiac complications. It is caused by loss-of-function mutations in the DMD gene coding for dystrophin, an essential protein involved in maintaining the structural integrity and function of muscle fibres. Eteplirsen was first approved by the FDA in September 2016 for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene, which codes for dystrophin, that is amenable to exon 51 skipping. Eteplirsen directly works on the DMD gene to promote dystrophin production. Eteplirsen was the first treatment for DMD approved by the FDA.
- Indication
Eteplirsen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with eteplirsen.
- Associated Conditions
- Duchenne Muscular Dystrophy (DMD)
- Associated Therapies
- -
Nippon Shinyaku’s DMD drug fails confirmatory trial
Nippon Shinyaku’s DMD therapy Viltepso faces uncertainty after failing a confirmatory trial, missing its primary objective and not distinguishing from placebo in patient mobility tests. Despite prior confidence in its efficacy, continued approval hinges on further clinical benefit verification. Meanwhile, Sarepta’s gene therapies, including Elevidys, are emerging as potential successors in DMD treatment.
FDA's Accelerated Approval Pathway Drives Momentum for Intractable, Fatal Diseases
Accelerated approval, despite recent controversies, has historically provided therapeutic momentum, particularly in HIV and cancer treatments. Key issues include reliance on surrogate endpoints and confirmatory trial failures, yet the pathway has led to significant drug approvals, offering hope for diseases with high unmet needs.
Six antisense oligonucleotide companies shaping the future of genetic medicine
Six companies—Ionis Pharmaceuticals, Isarna Therapeutics, Regulus Therapeutics, Sarepta Therapeutics, Secarna Pharmaceuticals, and Wave Life Sciences—are leading in the development of antisense oligonucleotides, a targeted treatment for various diseases. Ionis, a pioneer with over 40 drugs in its pipeline, recently priced a $500 million IPO. Isarna's ISTH0036 targets TGF-β for ophthalmic conditions. Regulus focuses on microRNA targeting with its lead candidate RGLS8429 for ADPKD. Sarepta has three approved PPMO therapies for DMD and a recent $1 billion deal with Arrowhead Pharmaceuticals. Secarna's SECN-15 targets NRP1 for oncology. Wave Life Sciences' WVE-003, for Huntington's disease, recently reported positive phase 1b/2a trial results. The antisense oligonucleotide market is projected to grow to $5,519 million by 2033.
Sarepta scraps a Duchenne drug as gene therapy sales rise
Sarepta Therapeutics discontinues SRP-5051 for Duchenne muscular dystrophy due to safety concerns and FDA feedback, as Elevidys gene therapy sales surge to $181 million in Q3. Elevidys revenue expected to exceed $2 billion next year, overshadowing the end of SRP-5051 development.
Sarepta pulls plug on Duchenne exon-skipping drug
Sarepta Therapeutics has halted development of vesleteplirsen, a more potent version of its DMD therapy Exondys 51, citing risk-benefit considerations, FDA feedback, and the evolving DMD therapeutic landscape. Elevidys, Sarepta's gene therapy, is rapidly gaining ground, with $181 million in Q3 sales compared to $249 million for Exondys 51 and its counterparts.
Sarepta Therapeutics, Inc. SEC 10-Q Report
Sarepta Therapeutics' Q3 2023 Form 10-Q highlights financial growth, key business developments, strategic initiatives, and emerging challenges. Notable financial metrics include $1,243.6 million in total revenues, $1,056.8 million in gross profit, $56.4 million in operating income, and $76.2 million in net income. Business highlights include FDA approvals for four products targeting Duchenne muscular dystrophy, new product launches, and ongoing clinical trials. Strategic initiatives focus on manufacturing expansion and capital management. Challenges include market risk, regulatory compliance, reimbursement uncertainty, and global expansion difficulties.
Duchenne Muscular Dystrophy (DMD) Pipeline Analysis, 2024
DelveInsight's 'Duchenne Muscular Dystrophy (DMD) Pipeline Insight, 2024' analyzes over 75 pipeline drugs from 75+ companies, focusing on recent FDA, EMA, and PMDA approvals, clinical trials, emerging therapies, and key players like Roche, Santhera, and Sarepta. The report covers various stages of development, routes of administration, and mechanisms of action, offering insights into the evolving DMD therapeutics landscape.
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
EMBARK trial (SRP-9001-301) assessed delandistrogene moxeparvovec safety/efficacy in DMD patients aged 4-7. Conducted at 42 sites, it was a phase 3, two-part, multinational, randomized, double-blind, placebo-controlled trial. Patients received either delandistrogene moxeparvovec or placebo, with crossover in Part 2. Primary endpoint was change in NSAA total score from baseline to week 52. Key secondary endpoints included micro-dystrophin expression at week 12, and changes in TTR and 10MWR from baseline to week 52.
Six biotechs driving progress in Duchenne muscular dystrophy
Six clinical-stage biotech companies are advancing Duchenne muscular dystrophy treatments: Wave Life Sciences, Sarepta Therapeutics, Capricor Therapeutics, Edgewise Therapeutics, Italfarmaco, and Avidity Biosciences. These companies focus on various therapeutic approaches, including RNA medicines, gene therapies, myosin inhibitors, and HDAC inhibitors. The global Duchenne treatment market is expected to grow significantly, driven by regulatory approvals and ongoing research.
DYNE-251 led to motor function improvements in DMD boys
Treatment with DYNE-251, Dyne Therapeutics' exon 51-skipping therapy, improved motor function in boys with Duchenne muscular dystrophy (DMD) in a Phase 1/2 trial. DYNE-251 increased dystrophin levels, with 20 mg/kg doses reaching 3.71% of normal, significantly higher than Exondys 51. Functional benefits were observed across various assessments, meeting European Medicines Agency thresholds for clinical significance. Dyne plans to initiate registrational cohorts and pursue expedited approval pathways, with an update expected by year's end.
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