US-based biopharmaceutical company Harmony Biosciences has secured a US Food and Drug Administration (FDA) orphan drug designation for its drug pitolisant, an investigational treatment for Prader-Willi Syndrome (PWS). 

Harmony now plans to advance the candidate into a Phase III clinical trial. The planned registrational TEMPO study will assess the safety and efficacy of pitolisant in treating excessive daytime sleepiness and behavioural disturbances seen in PWS. Harmony has announced that it aims to initiate the global study in Q1 2024, enrolling patients six years and older with PWS. 

Commercialised as Wakix in the US, pitolisant is approved by the Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy, a sudden muscle weakness that occurs while a person is awake, in adult patients with narcolepsy.  

The selective histamine 3 (H3) receptor antagonist/inverse agonist was initially developed by France-based Bioprojet, which granted an exclusive licence to Harmony for the development, manufacturing, and commercialisation of pitolisant in the US in 2017. 

Harmony reported Wakix sales of $582m for 2023, in its annual report. According to GlobalData’s Pharma Intelligence Center, pitolisant will generate nearly $2bn in sales in 2029, potentially being driven by an increase in demand following a possible approval for PWS. 

GlobalData is the parent company of Pharmaceutical Technology. 

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The orphan drug designation follows the announcement of preliminary positive Phase II (NCT04257929) data in 2022, where the trial met its primary endpoint of evaluating the variation on the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) to judge the therapy’s impact on EDS. 

PWS is a complex genetic condition that affects many parts of the body. It’s characterised by weak muscle tone, feeding difficulties, delayed development, and other issues. One of the most prominent features is an insatiable appetite, which can lead to chronic overeating and obesity if not managed carefully. 

In the announcement accompanying the orphan drug designation, Harmony’s chief medical officer Kumar Budur said: “We are excited about our upcoming Phase III TEMPO study and the progress we have made to broaden the clinical utility of pitolisant not just in Prader-Willi Syndrome but other rare diseases as part of our life cycle management programmes that, if successful, could potentially help over 100,000 patients.” 

Last May, Spanish biopharmaceutical company Palobiofarma teamed up with the Foundation for Prader Willi Research (FPWR) to develop its PWS drug PBF-999. The candidate is currently being investigated in a Phase II clinical trial.  

Note: This third paragraph has been updated to reflect the correct approved indication for Wakix. A previous version said the drug was approved for a different condition.