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

 
• By Derrick Gingery

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.

Hourglass
Many DMD patients are hoping time does not run out on their access to Elevidys. • Source: Shutterstock

Suddenly, birthdays have taken on much more importance among Duchenne muscular dystrophy patients. The difference between age five and age six could mean the difference between receiving the latest gene therapy and not ... at least for now.

Sarepta Therapeutics, Inc.’s recently launched gene therapy Elevidys (delandistrogene moxeparvovec-rokl) is indicated only for ambulatory four- and five-year-olds with DMD

Key Takeaways

  • Early Elevidys demand is among patients who soon will age out, but is expected to normalize.

  • Payer decisions and hospital cost negotiations add to fears some patients will not get the gene therapy in time

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