The FDA maintains its approval for the narcolepsy treatment in adults and recently expanded use in pediatric patients.


Summary: The FDA has denied a citizen petition filed by a short seller challenging the safety and effectiveness of Wakix (pitolisant) for treating excessive daytime sleepiness and cataplexy in adults with narcolepsy. The FDA reaffirmed Wakix’s favorable benefit-risk profile and maintained its recent approval for pediatric use.

Key Takeaways:

  • FDA Denial of Petition: The FDA rejected a petition challenging Wakix’s safety and effectiveness, reaffirming the drug’s favorable benefit-risk profile for treating narcolepsy.
  • Expansion to Pediatric Use: The FDA’s decision follows the recent approval of Wakix for treating excessive daytime sleepiness in pediatric patients aged 6 and older.
  • Short Seller’s Claims Dismissed: The petition, filed by a short seller, was deemed unfounded, with the FDA stating that the provided information did not alter its assessment of Wakix’s safety and effectiveness.

Harmony Biosciences announced that the US Food and Drug Administration (FDA) has denied a citizen petition filed by a short seller challenging the safety and effectiveness of its Wakix (pitolisant) for treating excessive daytime sleepiness and cataplexy in adults with narcolepsy.

The petition argued that pitolisant “poses a grave danger to patients” and was approved based “entirely on low-quality foreign clinical trials with numerous methodological red flags.” The petition requested that the FDA withdraw approval of Wakix for all indications, require Harmony to distribute an immediate alert to prescribers, and transition Wakix to a Compassionate Use Program with a risk evaluation and mitigation strategy protocol. 

The FDA denied the requests. “The FDA determined that Wakix has a favorable benefit-risk profile under its approved conditions of use, and your petition did not provide information that changes that assessment,” the denial letter states. 

It continues, “The FDA has carefully considered the information submitted in the petition, other data available to the agency, and relevant published literature. Based on our review of these materials and for the reasons stated, the petition is denied.”

Recently Approved for Pediatric Patients

The FDA denied the petition after approving a supplemental New Drug Application for Wakix on June 21, which expanded the indication to include the treatment of excessive daytime sleepiness in pediatric patients 6 years of age and older with narcolepsy. 

“We are pleased with the FDA’s decision to deny the short seller petition filed in March 2023, which tried to cast doubt on a safe and effective treatment while deliberately attempting to impact our stock price for profit,” reads a statement from Harmony Biosciences. “We appreciate the agency’s careful review of the unfounded claims in the petition and its definitive action to deny the claims and resolve any potential doubt about the favorable benefit-risk profile of Wakix.”  

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