HARMONY — Wakix FDA Review (NDA211150, ORIG 2, 2020)

HARMONY · 2020 · FDA Review (openFDA)

Report summary

This clinical review evaluates Harmony Biosciences' NDA 211150 for Wakix (pitolisant) to treat cataplexy in adult narcolepsy patients, building on its prior approval for excessive daytime sleepiness (EDS). The review re-analyzed efficacy data from HARMONY 1 and HARMONY CTP trials, confirming pitolisant's effectiveness for EDS but finding insufficient evidence for cataplexy. Safety data from clinical trials and postmarketing surveillance revealed a potential hypersensitivity signal, leading to labeling change recommendations. Other serious adverse events included depression, seizure, and suicidal ideation, though the overall safety profile remained consistent with the original NDA. Common adverse events were headache, insomnia, and nausea. While some psychiatric and cardiovascular events occurred, no new safety risks were identified from EU postmarketing data.

Key findings — cited to the page

HBS-101-CL-001 TEAE: Irritability	2 %	HBS-101-CL-001 · p.9
Irritability incidence (Pitolisant)	5 %	HARMONY I, HARMONY CTP, HARMONY I-bis · p.169
Irritability incidence (Placebo)	2 %	HARMONY I, HARMONY CTP, HARMONY I-bis · p.169
Irregular heart rate	1 patient	HARMONY I, HARMONY CTP, HARMONY I-bis · p.165
Irritability	1.5 %	HARMONY I-bis (P09-15) · p.163
Irritability	3.3 %	HARMONY I, HARMONY CTP, HARMONY I-bis (Unweighted) · p.164
Irritability	4.5 %	HARMONY I-bis (P09-15) · p.150
irritability incidence vs placebo	3.3 %	· p.45
Irritability	25.0 %	HARMONY CTP (P11-05) · p.149
Heart rate irregular	4.5 %	HARMONY I-bis (P09-15) · p.150
Irritability	5.6 %	HARMONY CTP (P11-05) · p.162
irritability incidence (placebo)	1.8 %	· p.45

Source: HARMONY ↗ · License: US Government public domain. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →