PLYMOUTH
MEETING, Pa., Sept. 7,
2023 /PRNewswire/ -- Harmony Biosciences
Holdings, Inc. ("Harmony") (Nasdaq: HRMY), a pharmaceutical company
dedicated to developing and commercializing innovative therapies
for patients with rare neurological diseases, today announced that
the U.S. Food and Drug Administration (FDA) has granted Orphan Drug
designation to pitolisant for the treatment of idiopathic
hypersomnia (IH).
The FDA's Orphan Drug designation incentivizes the advancement
of promising therapies for rare diseases. Approximately 80,000
people in the U.S. are believed to be affected by IH, with 40,000
currently having been diagnosed. IH is a condition with high unmet
medical need.
Harmony is currently evaluating the efficacy and safety of
pitolisant in adult patients with IH in the Phase 3 registrational
INTUNE study, a double-blind, placebo-controlled, randomized
withdrawal study. Topline study results are anticipated in the
fourth quarter of 2023 following enrollment completion nine months
ahead of plan.
"The FDA's decision to grant Orphan Drug designation reinforces
our belief in pitolisant as a promising therapy for adult patients
with IH, with the unique added benefit of it being a non-scheduled,
once-daily treatment option working through histamine to improve
wakefulness," said Kumar Budur,
M.D., Chief Medical Officer at Harmony Biosciences. "With the
completion of enrollment in our INTUNE study nine months ahead of
schedule and topline results expected in Q4, this designation is a
significant advance in our clinical and commercial development
initiatives. We look forward to working closely with the FDA and
the broader IH community to address the unmet medical needs of
patients living with this condition."
About Idiopathic Hypersomnia
Idiopathic Hypersomnia
(IH) is a rare and chronic neurological disease that is
characterized by excessive daytime sleepiness (EDS) despite
sufficient or even long sleep time. EDS in IH is usually not
alleviated by naps, longer sleep or more efficient sleep. People
living with IH experience significant EDS along with the symptoms
of sleep inertia (prolonged difficulty waking up from sleep) and
'brain fog' (impaired cognition, attention, and alertness). The
cause of IH is unknown, but it is likely due to alterations in
areas of the brain that stabilize states of sleep and wakefulness.
IH is one of the central disorders of hypersomnolence and, like
narcolepsy, is a debilitating sleep disorder that can result in
significant disruption in daily functioning.
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