Prescription Drug User Fee Act date is June 21, 2024
PLYMOUTH MEETING, Pa.,
Feb. 21, 2024 /PRNewswire/ -- Harmony
Biosciences Holdings, Inc. (Nasdaq: HRMY) announced that the U.S.
Food and Drug Administration (FDA) granted priority review for its
supplemental New Drug Application (sNDA) for WAKIX®
(pitolisant) tablets for the treatment of excessive daytime
sleepiness (EDS) or cataplexy in pediatric patients 6 years of age
and older with narcolepsy. The FDA has set a Prescription Drug User
Fee Act (PDUFA), or target action date, of June 21, 2024.
"The FDA's decision highlights the pressing need for new
treatment options for children living with narcolepsy and expedites
the timeline for its decision," said Jeffrey M. Dayno, M.D., President and Chief Executive Officer at
Harmony Biosciences. "We are committed to expanding the utility of
pitolisant for patients with high unmet medical needs and look
forward to collaborating with the FDA to potentially introduce a
new, non-scheduled treatment option for children with
narcolepsy."
The FDA accepted the sNDA and granted priority review based on a
Phase 3 multicenter, randomized, placebo-controlled study conducted
by Bioprojet that evaluated the safety and efficacy of pitolisant
in pediatric patients 6 to < 18 years with narcolepsy with or
without cataplexy. Based on the results of this study, Bioprojet
received approval from the European Medicines Agency last year
extending the indication for pitolisant to include the treatment of
narcolepsy in children aged 6 years and older, with or without
cataplexy.
About Narcolepsy
Narcolepsy is a rare, chronic,
debilitating neurological disease of sleep-wake state instability
that impacts approximately 170,000 Americans and is primarily
characterized by excessive daytime sleepiness (EDS) and cataplexy –
its two cardinal symptoms – along with other manifestations of REM
sleep dysregulation (hallucinations and sleep paralysis), which
intrude into wakefulness. EDS is the inability to stay awake and
alert during the day and is the symptom that is present in all
people living with narcolepsy. In most patients, narcolepsy is
caused by the loss of hypocretin/orexin, a neuropeptide in the
brain that supports sleep-wake state stability. This disease
affects men and women equally, with typical symptom onset in
adolescence or young adulthood; however, it can take up to a decade
to be properly diagnosed.
About WAKIX® (pitolisant)
Tablets
WAKIX, a first-in-class medication, is approved by
the U.S. Food and Drug Administration for the treatment
of excessive daytime sleepiness or cataplexy in adult patients with
narcolepsy and has been commercially available in
the U.S. since Q4 2019. It was granted orphan drug
designation for the treatment of narcolepsy in 2010, and
breakthrough therapy designation for the treatment of cataplexy in
2018. WAKIX is a selective histamine 3 (H₃) receptor
antagonist/inverse agonist. The mechanism of action of WAKIX is
unclear; however, its efficacy could be mediated through its
activity at H₃ receptors, thereby increasing the synthesis and
release of histamine, a wake promoting neurotransmitter. WAKIX was
designed and developed by Bioprojet (France). Harmony has an exclusive license
from Bioprojet to develop, manufacture and commercialize
pitolisant in the United
States.
INDICATIONS AND USAGE
WAKIX is indicated for the
treatment of excessive daytime sleepiness or cataplexy in adult
patients with narcolepsy.
IMPORTANT SAFETY INFORMATION
Contraindications
WAKIX is contraindicated in patients
with known hypersensitivity to pitolisant or any component of the
formulation. Anaphylaxis has been reported. WAKIX is also
contraindicated in patients with severe hepatic impairment.
Warnings and Precautions
WAKIX prolongs the QT
interval; avoid use of WAKIX in patients with known QT prolongation
or in combination with other drugs known to prolong the QT
interval. Avoid use in patients with a history of cardiac
arrhythmias, as well as other circumstances that may increase the
risk of the occurrence of torsade de pointes or sudden death,
including symptomatic bradycardia, hypokalemia or hypomagnesemia,
and the presence of congenital prolongation of the QT interval.
The risk of QT prolongation may be greater in patients with
hepatic or renal impairment due to higher concentrations of
pitolisant; monitor these patients for increased QTc. Dosage
modification is recommended in patients with moderate hepatic
impairment and moderate or severe renal impairment (see full
prescribing information). WAKIX is not recommended in patients with
end-stage renal disease (ESRD).
Adverse Reactions
In the placebo-controlled clinical
trials conducted in patients with narcolepsy with or without
cataplexy, the most common adverse reactions (≥5% and at least
twice placebo) for WAKIX were insomnia (6%), nausea (6%), and
anxiety (5%). Other adverse reactions that occurred at ≥2% and more
frequently than in patients treated with placebo included headache,
upper respiratory tract infection, musculoskeletal pain, heart rate
increased, hallucinations, irritability, abdominal pain, sleep
disturbance, decreased appetite, cataplexy, dry mouth, and
rash.
Drug Interactions
Concomitant administration of WAKIX
with strong CYP2D6 inhibitors increases pitolisant exposure by
2.2-fold. Reduce the dose of WAKIX by half.
Concomitant use of WAKIX with strong CYP3A4 inducers decreases
exposure of pitolisant by 50%. Dosage adjustments may be required
(see full prescribing information).
H1 receptor antagonists that cross the blood-brain barrier may
reduce the effectiveness of WAKIX. Patients should avoid centrally
acting H1 receptor antagonists.
WAKIX is a borderline/weak inducer of CYP3A4. Therefore, reduced
effectiveness of sensitive CYP3A4 substrates may occur when used
concomitantly with WAKIX. The effectiveness of hormonal
contraceptives may be reduced when used with WAKIX and
effectiveness may be reduced for 21 days after discontinuation of
therapy.
Use in Specific Populations
WAKIX may reduce the
effectiveness of hormonal contraceptives. Patients using hormonal
contraception should be advised to use an alternative non-hormonal
contraceptive method during treatment with WAKIX and for at least
21 days after discontinuing treatment.
There is a pregnancy exposure registry that monitors pregnancy
outcomes in women who are exposed to WAKIX during pregnancy.
Patients should be encouraged to enroll in the WAKIX pregnancy
registry if they become pregnant. To enroll or obtain information
from the registry, patients can call 1-800-833-7460. The safety and
effectiveness of WAKIX have not been established in patients less
than 18 years of age.
WAKIX is extensively metabolized by the liver. WAKIX is
contraindicated in patients with severe hepatic impairment. Dosage
adjustment is required in patients with moderate hepatic
impairment.
WAKIX is not recommended in patients with end-stage renal
disease. Dosage adjustment of WAKIX is recommended in patients with
moderate or severe renal impairment.
Dosage reduction is recommended in patients known to be poor
CYP2D6 metabolizers; these patients have higher concentrations of
WAKIX than normal CYP2D6 metabolizers.
Please see the Full Prescribing Information for
WAKIX for more information.
To report suspected adverse reactions, contact Harmony
Biosciences at 1-800-833-7460 or the FDA at 1-800-FDA-1088
or www.fda.gov/medwatch.
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 Investor Contact:
Luis Sanay, CFA
445-235-8386
lsanay@harmonybiosciences.com
Harmony Biosciences Media Contact:
Cate McCanless
202-641-6086
cmccanless@harmonybiosciences.com
View original content to download
multimedia:https://www.prnewswire.com/news-releases/us-food-and-drug-administration-grants-priority-review-to-harmony-biosciences-application-for-wakix-pitolisant-in-pediatric-narcolepsy-302066778.html
SOURCE Harmony Biosciences