HARMONY BIOSCIENCES RECEIVES U.S. FOOD AND DRUG ADMINISTRATION ORPHAN DRUG DESIGNATION FOR PITOLISANT IN PRADER-WILLI SYNDROME

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Harmony Biosciences Receives FDA Orphan Drug Designation for Pitolisant in Prader-Willi Syndrome Treatment

Insights

Medical Research Analyst

The FDA's Orphan Drug designation for pitolisant represents a significant milestone for Harmony Biosciences, potentially addressing a rare and complex condition like Prader-Willi syndrome (PWS). The designation is not only a testament to the therapeutic potential of pitolisant but also a strategic advantage for Harmony in terms of financial incentives and market exclusivity.

From a research perspective, the upcoming Phase 3 TEMPO study is crucial. The study's design as a randomized, double-blind, placebo-controlled trial is the gold standard for assessing drug efficacy and safety. The focus on both excessive daytime sleepiness (EDS) and behavioral disturbances addresses the multifaceted nature of PWS, which may enhance the drug's value proposition if the outcomes are positive.

Given the prevalence of PWS in the U.S., the successful development of pitolisant could meet a significant unmet medical need. However, the success hinges on the drug's performance in the Phase 3 trials and the subsequent FDA approval process.

Financial Analyst

Harmony Biosciences' stock may see increased investor interest following the Orphan Drug designation, as it indicates regulatory support and potential for market exclusivity post-approval. The financial benefits, including tax credits and user fee waivers, can improve Harmony's cost efficiency during the development phase.

However, investors should consider the inherent risks of clinical development. The outcome of the Phase 3 TEMPO study will be a pivotal factor in Harmony's financial trajectory. Positive results could lead to a surge in the company's valuation, while negative outcomes could have the opposite effect. The seven-year market exclusivity, if granted upon approval, could provide a temporary monopoly, allowing Harmony to set a premium price for pitolisant, thus improving profit margins.

Long-term, Harmony's commitment to addressing unmet medical needs in the PWS community could foster brand loyalty and potentially open doors to additional indications for pitolisant, further expanding its market potential.

Healthcare Economist

The Orphan Drug designation for pitolisant has broader implications beyond Harmony Biosciences. It underscores the FDA's commitment to facilitating drug development for rare diseases, which often struggle with funding and support due to a smaller patient population.

Economic incentives provided by the designation can significantly reduce the cost barriers associated with drug development. For the healthcare system, this can translate into savings if pitolisant proves to be an effective treatment, potentially reducing the long-term care costs associated with PWS.

However, the cost-effectiveness of pitolisant will be a point of interest, as orphan drugs often come with high price tags due to the significant investment required for their development and the need to recoup costs within a smaller patient market. Policymakers and payers will likely scrutinize the pricing and reimbursement strategies for pitolisant to ensure that it is accessible to the PWS population without undue financial burden on the healthcare system.

PLYMOUTH MEETING, Pa., Feb. 20, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisant for the treatment of Prader-Willi syndrome (PWS).

"The decision to grant Orphan Drug designation to pitolisant indicates that it could be a promising treatment option for people living with Prader-Willi syndrome," said Kumar Budur, M.D., M.S., Chief Medical Officer at Harmony Biosciences. "This designation marks an important step forward in our PWS development program and we are eager to continue working with the FDA and the broader community of PWS patients and caregivers to address their high unmet medical needs."

FDA Orphan Drug designation incentivizes the advancement of promising therapies for rare diseases by providing tax credits for clinical development, waivers for user fees, and seven years of market exclusivity following drug approval. Approximately 15,000 – 20,000 people in the U.S. are living with PWS, the majority experiencing behavioral symptoms and more than half with excessive daytime sleepiness (EDS).

In the upcoming Phase 3 registrational TEMPO study, Harmony will assess the safety and efficacy of pitolisant in treating EDS and behavioral disturbances in PWS. This global study, anticipated to begin in Q1 2024, will be a randomized, double-blind, placebo-controlled trial in patients six years and older with PWS.

Dr. Budur added, "We are excited about our upcoming Phase 3 TEMPO study and the progress we have made to broaden the clinical utility of pitolisant not just in PWS but other rare diseases as part of our life cycle management programs that, if successful, could potentially help over 100,000 patients. On behalf of Harmony, I would like to thank all the patients and family members for participating in our clinical trials, as well as our investigators and site personnel for their commitment to advancing science."

About Prader-Willi Syndrome

PWS is an orphan/rare, genetic neurological disorder with many of the symptoms resulting from hypothalamic dysfunction. The hypothalamus is the part of the brain that controls both sleep-wake state stability and signals that mediate the balance between hunger and satiety, resulting in the main symptoms in patients with PWS, hyperphagia (an intense persistent sensation of hunger accompanied by food preoccupations, an extreme drive to consume food, food-related behavior problems, and a lack of normal satiety), EDS and behavioral symptoms. Other features include low muscle tone, short stature, and cognitive impairment.

About Harmony Biosciences

At Harmony Biosciences, we specialize in developing and delivering treatments for rare neurological diseases that others often overlook. We believe that where empathy and innovation meet, a better life can begin for people living with neurological diseases. Established by Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth Meeting, PA, our team of experts from a wide variety of disciplines and experiences is driven by our shared conviction that innovative science translates into therapeutic possibilities for our patients, who are at the heart of everything we do. For more information, please visit www.harmonybiosciences.com. 

Forward Looking Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: our commercialization efforts and strategy for WAKIX; the rate and degree of market acceptance and clinical utility of WAKIX, pitolisant in additional indications, if approved, and any other product candidates we may develop or acquire, if approved; our research and development plans, including our development activities with Bioprojet, and plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; the availability of favorable insurance coverage and reimbursement for WAKIX; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any of our product candidates, including those we are developing with Bioprojet; our failure to achieve the potential benefits of the 2022 LCA with Bioprojet; our estimates regarding expenses, future revenue, capital requirements and needs for additional financing; our ability to identify additional products or product candidates with significant commercial potential that are consistent with our commercial objectives; our commercialization, marketing and manufacturing capabilities and strategy; significant competition in our industry; our intellectual property position; loss or retirement of key members of management; failure to successfully execute our growth strategy, including any delays in our planned future growth; our failure to maintain effective internal controls; the impact of government laws and regulations; volatility and fluctuations in the price of our common stock; the significant costs and required management time as a result of operating as a public company; the fact that the price of Harmony's common stock may be volatile and fluctuate substantially; statements related to our intended share repurchases and repurchase timeframe and the significant costs and required management time as a result of operating as a public company. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (the "SEC") on February 21, 2023, and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

Harmony Biosciences Media Contact: 
Cate McCanless 
202-641-6086 
cmccanless@harmonybiosciences.com 

Harmony Biosciences Investor Contact: 
Luis Sanay, CFA 
445-235-8386 
lsanay@harmonybiosciences.com  

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SOURCE Harmony Biosciences

FAQ

What is the significance of the FDA granting Orphan Drug designation to pitolisant for Prader-Willi syndrome?

The FDA Orphan Drug designation incentivizes the advancement of promising therapies for rare diseases like Prader-Willi syndrome by providing tax credits, fee waivers, and market exclusivity.

How many people in the U.S. are estimated to be living with Prader-Willi syndrome?

Approximately 15,000 – 20,000 people in the U.S. are living with Prader-Willi syndrome, with many experiencing behavioral symptoms and excessive daytime sleepiness.

What will the upcoming Phase 3 registrational TEMPO study by Harmony Biosciences focus on?

The Phase 3 TEMPO study will assess the safety and efficacy of pitolisant in treating excessive daytime sleepiness and behavioral disturbances in patients with Prader-Willi syndrome.