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'Surrogate Of A Surrogate' Not Good Enough For Raxone Accelerated Approval

 
• By Michael Cipriano

FDA’s decision to require second pre-approval trial for Santhera’s DMD candidate is just latest example of the inability of rare disease treatments with surrogate endpoints to qualify for pathway.

Rare disease candidates with surrogate endpoints are struggling to gain accelerated approval from FDA, most recently highlighted by the agency declining to grant Santhera Pharmaceuticals accelerated approval for its Duchene muscular dystrophy candidate Raxone (idebenone).

The decision will force the Swiss drugmaker to conduct another Phase III study of the drug, for which it expects...

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