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