PLYMOUTH MEETING, Pa. - Harmony Biosciences Holdings , Inc. (NASDAQ: NASDAQ:HRMY), a pharmaceutical company, has announced the commencement of a global Phase 3 trial, named the TEMPO study, to assess the safety and efficacy of pitolisant for treating excessive daytime sleepiness (EDS) and behavioral symptoms in individuals aged six and older with Prader-Willi syndrome (PWS).
The study follows a successful End-of-Phase 2 meeting with the U.S. Food & Drug Administration (FDA), which also recently granted Orphan Drug Designation (ODD) to pitolisant for PWS treatment. This designation aims to facilitate the drug's development by offering various incentives, including seven years of market exclusivity post-approval.
Pitolisant, currently marketed in the United States under the brand name WAKIX®, is an FDA-approved treatment for EDS or cataplexy in adults with narcolepsy. However, it is not yet approved for use in PWS patients and is being investigated for this purpose.
The TEMPO study is a randomized, double-blind, placebo-controlled, multicenter trial with an open-label extension period. "With the initiation of this study and the FDA's recent decision to grant ODD for pitolisant in PWS, we aim to potentially introduce a new, non-scheduled treatment option for EDS and the common behavioral symptoms in patients living with this condition," stated Dr. Kumar Budur, Chief Medical Officer of Harmony Biosciences.
PWS is a rare genetic disorder characterized by a constant state of hunger, sleep disturbances, and behavioral issues, among other symptoms. An estimated 15,000 to 20,000 people in the U.S. have PWS, with the majority experiencing behavioral symptoms and over half suffering from EDS. Currently, there is no FDA-approved treatment specifically for EDS in this patient population.
Harmony Biosciences is actively expanding the clinical applications of pitolisant to address unmet medical needs in PWS and other rare diseases. "These efforts are integral to our life cycle management programs, which, if successful, could benefit over 100,000 patients," added Dr. Budur.
The information in this article is based on a press release statement.
InvestingPro Insights
As Harmony Biosciences Holdings, Inc. (NASDAQ: HRMY) embarks on its Phase 3 trial for pitolisant, the company's financial health and market performance offer additional context for investors following its clinical developments. According to InvestingPro data, Harmony Biosciences boasts an adjusted market cap of $1.86 billion and has experienced a notable revenue growth of 32.93% over the last twelve months as of Q1 2023. This growth is reflective of the company's strategic initiatives and its efforts to expand the indications of its leading product, WAKIX®.
InvestingPro Tips suggest that Harmony's management has been proactive in enhancing shareholder value through aggressive share buybacks. Additionally, the company's solid free cash flow yield, as implied by its valuation, and the fact that its liquid assets exceed short-term obligations, provide a cushion for the company as it invests in clinical trials and research. These financial measures are crucial for Harmony as it navigates the costly and time-intensive process of drug development and regulatory approval.
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