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Sarepta Winning Payer War Over Duchenne Muscular Dystrophy Drug Coverage

Executive Summary

Exondys 51 has been met with recommendations for restrictions by many insurers, but after consultations with experts and Sarepta execs coverage is expanding.

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Elevidys Payer Gauntlet Complicated By Fear Patients Could Age Out Of DMD Gene Therapy’s Label

Launching much faster than it told a US FDA advisory committee that it would, Sarepta is focusing on Duchenne muscular dystrophy patients about to turn six in the initial roll-out of its gene therapy Elevidys amid fears the wait for payer authorization could mean losing access to the treatment.

Elevidys’ Restricted Label Helps Sarepta Pressure Payers For Coverage Of DMD Gene Therapy

Sarepta says the age limit in the indication could encourage swifter insurance coverage of the newly approved gene therapy for Duchenne muscular dystrophy so potential patients don't age out of the treatment window before they would be eligible for reimbursement.

Unpredictable DMD Progression Complicates Sarepta’s Gene Therapy Efficacy Claims

As with many rare diseases, a clear natural history would make deciphering the data easier, but for now what Sarepta characterizes as stabilization is seen by FDA reviewers as expected improvement based on previous measures of disease progression at younger ages.

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