Inside A Phase III Disappointment: Santhera Reduced Target Population, Now Needs Another Trial
FDA seemed on board with Swiss drugmaker's plans for study of Duchenne muscular dystrophy candidate Raxone, but didn't look too kindly on firm's decision to drop subjects using concomitant glucocorticoids.
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Keeping Track: Janssen Gets A Breakthrough Therapy Status, FDA Awards Two Orphan Drug Designations, Portola Lands A CRL
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Santhera Setback Could Have Wide-Reaching Effects For DMD Drugs
The DMD community faces another setback as FDA pushes back timelines for Santhera and its respiratory drug, pushing the market to question what will come for other DMD treatments in development.
'Surrogate Of A Surrogate' Not Good Enough For Raxone Accelerated Approval
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.