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Retrophin's Focus Shifts After Phase III PKAN Drug's Failure

 
• By Joseph Haas

Fosmetpantotenate missed all endpoints in a Phase III trial, so Retrophin will shelve the candidate for the rare disease PKAN. Now, the biotech turns focus to Phase III sparsentan in a pair of indications.

Child-Sorrow
Retrophin misses its Phase III endpoint in PKAN, which typically manifests in childhood

Retrophin Inc. will shelve its most advanced pipeline asset and place its R&D focus on sparsentan in focal segmental glomerulosclerosis (FSGS) and IgA nephropathy (IgAN), after announcing on 22 August that fosmetpantotenate (fosmet) failed an 84-patient Phase III trial in the rare central nervous system disorder pantothenate kinase-associated neurodegeneration (PKAN).

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