BioMarin Gets Unfavorable View From Panel on Duchenne Drug
- Panel voted failed final-phase trial weakens earlier studies
- Sarepta is developing a related drug for the rare disease
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BioMarin Pharmaceutical Inc.’s experimental drug for Duchenne muscular dystrophy got an unfavorable review from U.S. regulatory advisers, possibly hurting its chances of becoming the first therapy on the market for the genetic disease that affects mainly young boys.
A panel of outside Food and Drug Administration advisers voted at a meeting Tuesday that inconsistencies in clinical trials of the drug weakened BioMarin’s argument that the treatment would work.