Exondys 51 (eteplirsen) vs Amondys 45 (casimersen)
Exondys 51 (eteplirsen) vs Amondys 45 (casimersen)
Exondys 51 (eteplirsen) and Amondys 45 (casimersen) are both antisense oligonucleotides approved for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation amenable to exon 51 or exon 45 skipping, respectively. Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in the exclusion of this exon during mRNA processing, while casimersen targets exon 45 for skipping. The choice between these two medications would depend on the specific exon where the patient's mutation is located; genetic testing can determine whether a patient's DMD is amenable to exon 51 or exon 45 skipping therapy.
Difference between Exondys 51 and Amondys 45
| Metric | Exondys 51 (eteplirsen) | Amondys 45 (casimersen) |
|---|---|---|
| Generic name | Eteplirsen | Casimersen |
| Indications | Treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene amenable to exon 51 skipping | Treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene amenable to exon 45 skipping |
| Mechanism of action | Antisense oligonucleotide directed to exon 51 of dystrophin pre-mRNA, resulting in skipping of this exon during mRNA processing and an abbreviated dystrophin protein | Antisense oligonucleotide directed to exon 45 of dystrophin pre-mRNA, resulting in skipping of this exon during mRNA processing and an abbreviated dystrophin protein |
| Brand names | Exondys 51 | Amondys 45 |
| Administrative route | Intravenous (IV) | Intravenous (IV) |
| Side effects | Balance disorder, vomiting, rash, and renal impairment | Upper respiratory tract infections, cough, fever, headache, joint pain, and throat pain |
| Contraindications | Hypersensitivity to eteplirsen or any of the excipients | Hypersensitivity to casimersen or any of the excipients |
| Drug class | Antisense oligonucleotide | Antisense oligonucleotide |
| Manufacturer | Sarepta Therapeutics, Inc. | Sarepta Therapeutics, Inc. |
Efficacy
Efficacy of Exondys 51 (Eteplirsen)
Exondys 51 (eteplirsen) is an antisense oligonucleotide designed for the treatment of Duchenne Muscular Dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene amenable to exon 51 skipping. This medication is intended to bind to exon 51 of dystrophin pre-mRNA, resulting in the exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. The aim is to enable the production of an internally truncated but functional dystrophin protein.
Clinical trials assessing the efficacy of Exondys 51 have shown mixed results. The approval of eteplirsen by the FDA was based on the increase in dystrophin production in skeletal muscle observed in some patients treated with the drug. However, the clinical benefit of such dystrophin production, such as improvement in motor function, has not been conclusively demonstrated in controlled trials. The FDA's accelerated approval was contingent upon the sponsor's commitment to conduct further clinical trials to verify the drug's clinical benefit.
Efficacy of Amondys 45 (Casimersen)
Amondys 45 (casimersen) is another antisense oligonucleotide therapy approved for the treatment of DMD in patients with a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. Similar to eteplirsen, casimersen is designed to bind to exon 45 of the dystrophin pre-mRNA, leading to exclusion of this exon during mRNA processing and production of an internally truncated dystrophin protein that is expected to have some functionality.
The approval of Amondys 45 was also based on the surrogate endpoint of an increase in dystrophin production in skeletal muscle in patients treated with the drug. The clinical trials demonstrated a modest increase in dystrophin levels, but the clinical significance of this finding has yet to be confirmed. As with eteplirsen, the FDA has required further clinical studies to establish the clinical benefit of Amondys 45, including assessments of motor function and quality of life in patients receiving the therapy.
Regulatory Agency Approvals
Exondys 51
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Food and Drug Administration (FDA), USA
Amondys 45
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Food and Drug Administration (FDA), USA
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If Exondys 51 or Amondys 45 are not approved or available in your country (e.g. due to supply issues), you can access them via Everyone.org.
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