FDA Refusal to File Wakix sNDA

FDA has issued a refusal to file letter for Harmony Biosciences’ Wakix (pitolisant) to treat excessive daytime sleepiness in adults with idiopathic hypersomnia. A company statement says the company plans to continue with a pivotal Phase 3 study based on FDA input. “We believe this study design will effectively demonstrate the benefit of pitolisant in patients with idiopathic hypersomnia based on the robust clinical response that was observed in the Phase 3 INTUNE study,” says Harmony Biosciences’ chief medical and scientific officer Kumar Budur.

Wakix is approved by FDA to treat excessive daytime sleepiness in adults and pediatric patients aged six and older with narcolepsy. It was granted orphan drug designation to treat narcolepsy in 2010 and breakthrough therapy designation to treat cataplexy in 2018. It is described as a selective histamine 3 receptor antagonist/inverse agonist.