A new controversy is emerging over a costly treatment for Duchenne muscular dystrophy, a rare disease that can send boys to an early death, thanks to a new analysis suggesting that clinical data is so limited that it is impossible to determine whether the medication is worth a price tag of $300,000 or more.

The medicine, which is called Exondys 51 and marketed by Sarepta Therapeutics (SRPT), caused a heated public debate over regulatory approval standards and satisfying unmet medical needs during the run-up to its endorsement nearly three years ago by the Food and Drug Administration.

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  • How dare ICER use the stated list price – ““many factors were ignored including mandatory discounts and rebates to insurers and pharmacy benefit companies.” This reads like another proof that this drug should never have been approved in the first place.

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