Product information and disease description Place of product in therapy Approaches to treatment Current treatment options and standard of care

Limitations of current therapies

3 evidence topics · 3 sources

Summary

Available treatments have different eligibility restrictions, administration requirements, and evidentiary limitations. Eteplirsen is restricted to exon 51-skipping-amenable mutations and requires weekly intravenous infusion; its labeling states that continued approval may depend on verification of clinical benefit. Delandistrogene moxeparvovec-rokl is limited to ambulatory individuals aged four years or older and carries a boxed warning for serious liver injury and acute liver failure, including fatal outcomes. The 2019 ICER assessment of eteplirsen and golodirsen is historical and is not an assessment of z-rostudirsen.

Historical assessment of eteplirsen and golodirsen

“In the absence of persuasive evidence demonstrating clinical effectiveness, ICER is unable to calculate value-based price benchmarks for eteplirsen or golodirsen.” (opens the source at this quote in a new tab)

Eteplirsen confirmatory-evidence requirement

“Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.” (opens the source at this quote in a new tab)