Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Amondys 45 · 1 trial · 1 indication
This will be assessed by the comparison of quantification of protein in muscle biopsy tissue by western blot from baseline to 1 year post-initiation of treatment.
This will be measured by capturing and reviewing Adverse Events as defined by CTCAE v4.0.
| Arm | Type | Description |
|---|---|---|
| AMONDYS 45 | EXPERIMENTAL | This arm will involve the treatment of boys with DMD who have a duplication of exon 45, for which AMONDYS 45 will target skipping of this exon. |
| EXONDYS 51 | EXPERIMENTAL | This arm will involve the treatment of boys with DMD who have a duplication of exon 51, for which EXONDYS 51 will target skipping of this exon. |
| VYONDYS 53 | EXPERIMENTAL | This arm will involve the treatment of boys with DMD who have a duplication of exon 53, for which VYONDYS 53 will target skipping of this exon. |
| Name | Type | Description |
|---|---|---|
| Amondys 45 | DRUG | This drug is used to target skipping of exon 45 of the dystrophin gene. |
| Exondys 51 | DRUG | This drug is used to target skipping of exon 51 of the dystrophin gene. |
| Vyondys 53 | DRUG | This drug is used to target skipping of exon 53 of the dystrophin gene. |
Inclusion Criteria: * Is a male with DMD and has an out-of-frame duplication of either exon 45, 51, or 53, with a normal copy number of all other DMD exons. * Is above age 6 months of age. * Has sufficient muscle mass in a pair of bilateral muscles that will allow for pre- and post-treatment muscle...
Amondys 45 is an investigational small molecule being developed for the treatment of Duchenne Muscular Dystrophy (DMD). It is being studied in patients who carry eligible DMD duplications. The drug is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
Amondys 45 is being developed by Sarepta Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol SRPT. The company is conducting clinical trials to evaluate the drug's efficacy and safety in patients with Duchenne Muscular Dystrophy.
Amondys 45 is currently in Phase 2 clinical development. It is an investigational drug and has not yet received regulatory approval. The drug is being studied in a completed Phase 2 trial that evaluated its efficacy and safety in patients with Duchenne Muscular Dystrophy.
Amondys 45 has been studied in one clinical trial with the identifier NCT04179409. This was a 48-week, open-label, Phase 2 study that evaluated the efficacy and safety of Amondys 45, along with other drugs, in subjects with Duchenne Muscular Dystrophy carrying eligible DMD duplications. The trial has been completed.
Amondys 45 is not the same as EXONDYS 51 or VYONDYS 53. These are three separate drugs that were evaluated together in a single clinical trial (NCT04179409) for the treatment of Duchenne Muscular Dystrophy. Each drug targets different genetic mutations in the DMD gene.