OSAKA, Japan & CAMBRIDGE, Mass. - Thursday, 06. August 2026
Adults Taking ORZEYFUL Experienced Significant and Meaningful
Improvements Across the Full Range of Symptoms of Narcolepsy Type 1
(NT1) in Clinical Trials Compared to Those on Placebo
As a First-in-Class Orexin Treatment, ORZEYFUL has the Potential to Redefine NT1 Care Beyond Individual Symptoms
Takeda is Advancing U.S. Launch Preparations and Expects to Make
ORZEYFUL Available Following Completion of the Drug Enforcement
Administration (DEA) Scheduling Process
(BUSINESS
WIRE)--Takeda (TSE:4502/NYSE:TAK) announced that the U.S. Food and Drug
Administration (FDA) approved ORZEYFUL™ (oveporexton), an oral orexin
receptor 2 (OX2R) agonist, for the treatment of narcolepsy type 1 (NT1,
narcolepsy with cataplexy) in adults.* The persistent 24-hour nature of
NT1 is driven by orexin deficiency and can severely impact people’s
lives. As a first-in-class orexin treatment, ORZEYFUL is the only
medicine indicated in the U.S. to treat the disease holistically rather
than individual symptoms.
“The FDA approval of ORZEYFUL marks a
new chapter for the narcolepsy type 1 community, as we introduce an
entirely new class of medicine that will potentially redefine how this
disease is managed and how people feel on treatment,” said Julie Kim,
president and chief executive officer of Takeda. “We are proud to have
discovered and developed this orexin treatment innovation and will bring
it to adults living with NT1 as quickly as possible.”
NT1 is a
rare, chronic neurological disease driven by a loss of orexin and
affects an estimated 120,000 people in the U.S. People with NT1
experience excessive daytime sleepiness, cataplexy (sudden loss of
muscle tone), cognitive symptoms and disrupted nighttime sleep that can
severely impact multiple aspects of daily life, including work,
education and social interactions.
"For those of us living with
narcolepsy type 1, symptoms reshape everyday life and extend far beyond
what most people understand about the condition," said Julie Flygare,
President and CEO at Project Sleep and a person with NT1. "ORZEYFUL’s
approval is a historic moment that expands our treatment choices and
gives me great hope for the future and for our community."
“Until
now, people have managed narcolepsy type 1 with treatments that target
symptom relief,” said Dr. Emmanuel Mignot, M.D., Ph.D., principal U.S.
investigator in the ORZEYFUL Phase 3 program. “As the first and only
approved orexin therapy to treat the broad spectrum of the disease,
ORZEYFUL can enable a different kind of conversation in the doctor’s
office about treatment options.”
The approval is based on a
comprehensive clinical program including the global Phase 3 FirstLight
(TAK-861-3001) and RadiantLight (TAK-861-3002) studies that showed
oveporexton offers statistically significant improvements across the
full range of symptoms of the disease. These included improvements in
excessive daytime sleepiness, cataplexy and health-related quality of
life. Oveporexton was generally well-tolerated with a safety profile
consistent across clinical studies to date. The most common side effects
include trouble sleeping (insomnia), urinary urgency, urinary frequency
and excessive saliva. Learn more about the Phase 3 data results here.
"With
this approval, we are turning scientific discovery into reality for
adults living with narcolepsy type 1," said Andy Plump, M.D., Ph.D.,
President, Research & Development, Takeda. "ORZEYFUL is just the
beginning. With orexin emerging as a powerful therapeutic pathway,
Takeda is unlocking the potential of this new class of medicine for
patients across a range of disorders.”
Next Steps for ORZEYFUL Availability
The
controlled substance classification for ORZEYFUL is currently under
review by the Drug Enforcement Administration (DEA) and is expected
within 90 days. After the controlled substance classification is
determined, ORZEYFUL will be made available through a specialty pharmacy
to U.S. healthcare providers and adults living with NT1. To sign up for
updates visit ORZEYFUL.com.
The FDA approval is not expected to
have a significant impact on the full year consolidated financial
forecast for the fiscal year ending March 31, 2027.
*For
information regarding the submission of the new drug application for
oveporexton to the U.S. FDA, please refer to Takeda’s press release
dated February 10, 2026, “U.S. Food and Drug Administration Accepts New
Drug Application and Grants Priority Review for Takeda’s Oveporexton
(TAK-861) as a Potential First-in-Class Therapy for Narcolepsy Type 1”.
About ORZEYFUL (oveporexton)
ORZEYFUL
(oveporexton) is an oral orexin receptor 2 (OX2R) agonist, which
selectively stimulates the OX2R to restore signaling and address the
underlying orexin deficiency associated with narcolepsy type 1 (NT1). By
activating OX2Rs, ORZEYFUL promotes wakefulness and reduces abnormal
rapid eye movement (REM)-sleep like phenomena, including cataplexy
(sudden and temporary loss of muscle tone), to address a range of
daytime and nighttime symptoms as evaluated in clinical studies and
consistent with the approved label.
INDICATION
ORZEYFUL is indicated for the treatment of narcolepsy type 1 (narcolepsy with cataplexy) in adult patients.
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
ORZEYFUL is contraindicated in patients taking strong CYP3A inhibitors.
WARNINGS AND PRECAUTIONS
Insomnia:
In pooled phase 3 studies in patients with NT1, 60%, 55%, and 1% of
patients in the ORZEYFUL 2 mg twice daily, ORZEYFUL 1 mg twice daily,
and placebo groups, respectively, developed insomnia. Prior to ORZEYFUL
treatment initiation, inform patients about the risk of insomnia at
initiation of treatment. If insomnia persists beyond 7 days and
significantly impacts daytime functioning or quality of life, consider
ORZEYFUL dosage reduction or discontinuation.
Urinary Frequency
and Urgency: ORZEYFUL may cause or worsen urinary frequency and urgency.
In pooled phase 3 studies in patients with NT1:
58%, 53%,
and 5% of patients in the ORZEYFUL 2 mg twice daily, ORZEYFUL 1 mg twice
daily, and placebo groups, respectively, developed urinary frequency.
16%, 15%, and 1% of patients in the ORZEYFUL 2 mg twice daily,
ORZEYFUL 1 mg twice daily, and placebo groups, respectively, developed
urinary urgency.
These lower urinary tract symptoms are
consistent with ORZEYFUL’s mechanism of action through agonism of the
orexin receptor 2 (OX2R) on central micturition pathways. Prior to
initiation of ORZEYFUL treatment, inform patients about the risk of
urinary frequency and urgency and screen for lower urinary tract
symptoms. Monitor ORZEYFUL-treated patients with a history of overactive
bladder for worsening urinary tract symptoms.
Creatine
Phosphokinase Elevations: In pooled phase 3 studies in patients with
NT1, asymptomatic creatine phosphokinase elevations >5x ULN were
observed in 11% (21/196) of ORZEYFUL-treated patients and 5% (4/76) of
placebo treated patients. Two of these cases were characterized by
markedly elevated CPK and transaminase levels; both patients
discontinued treatment. None of the cases were associated with
myoglobinuria or renal impairment. Advise patients to report unexplained
muscle pain, weakness, or dark urine, particularly when engaging in
vigorous physical activity or taking concomitant drugs associated with
myotoxicity.
ADVERSE REACTIONS
The most common adverse
reactions (incidence ≥5% and greater than placebo) reported in phase 3
studies with ORZEYFUL were insomnia, pollakiuria, micturition urgency,
and salivary hypersecretion.
DRUG INTERACTIONS
Concomitant use with strong or moderate CYP3A inhibitors increases
oveporexton exposures, which may increase the risk of
ORZEYFUL-associated adverse reactions
Concomitant use of strong
and moderate CYP3A inducers can increase oveporexton metabolism and
decrease plasma levels of oveporexton, which may decrease the
effectiveness of ORZEYFUL
USE IN SPECIFIC POPULATIONS
Pregnancy:
There is a pregnancy exposure registry that monitors pregnancy outcomes
in women who are exposed to ORZEYFUL during pregnancy. Patients should
be encouraged to enroll in the ORZEYFUL pregnancy registry if they
become pregnant. To enroll or obtain information from the registry,
patients can call 1-877-371-0309. Available data from clinical trials
with ORZEYFUL use during pregnancy are insufficient to identify a
drug-associated risk of major birth defects, miscarriage, or other
adverse maternal or fetal outcomes.
Lactation: There are no data
available on the presence of oveporexton in human milk, the effects on
the breastfed infant, or the effects on milk production. Animal studies
indicate that oveporexton was present in the milk of lactating rats.
When a drug is present in animal milk, it is likely that the drug will
be present in human milk.
Hepatic Impairment: Avoid use of
ORZEYFUL in patients with severe hepatic impairment (Child-Pugh C), as
it has not been studied in this population.
Renal Impairment:
Avoid use of ORZEYFUL in patients with severe renal impairment on
dialysis (eGFR <15 mL/minute), as it has not been studied in this
population.
DRUG ABUSE AND DEPENDENCE
ORZEYFUL contains
oveporexton. Controlled substance schedule to be determined after review
by the Drug Enforcement Administration.
ORZEYFUL has potential
for abuse and misuse. Carefully evaluate patients for a recent history
of drug abuse, especially those with a history of CNS stimulant, and
follow such patients closely, observing them for signs of misuse or
abuse of ORZEYFUL.
Important Note: Prescribing Information is subject to change pending DEA scheduling and final label publication.
Please click for Full Prescribing Information.
About Takeda’s Orexin Franchise
Takeda
is the leader in orexin science with a tailored portfolio of
investigational orexin agonists in pre-clinical and clinical stages for
multiple-sleep wake disorders and other indications where orexin plays a
role including respiration, mood and metabolism. ORZEYFUL (oveporexton)
is the lead orexin receptor 2 (OX2R) agonist asset in Takeda’s orexin
franchise and has been approved by regulatory bodies in China and the
United States for the treatment of narcolepsy type 1 (NT1). The company
is also investigating other oral orexin agonists, including TAK-360
being investigated for the treatment of NT1, narcolepsy type 2 (NT2) and
idiopathic hypersomnia (IH), as well as TAK-495.
About Takeda
Takeda
is focused on creating better health for people and a brighter future
for the world. We aim to discover and deliver life-transforming
treatments in our core therapeutic and business areas, including
gastrointestinal and inflammation, rare diseases, plasma-derived
therapies, oncology, neuroscience and vaccines. Together with our
partners, we aim to improve the patient experience and advance a new
frontier of treatment options through our dynamic and diverse pipeline.
As a leading values-based, R&D-driven biopharmaceutical company
headquartered in Japan, we are guided by our commitment to patients, our
people and the planet. Our employees in approximately 80 countries and
regions are driven by our purpose and are grounded in the values that
have defined us for more than two centuries. For more information, visit
www.takeda.com.
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Contacts
Investor Relations
Christopher O’Reilly
takeda.ir.contact@takeda.com
Media Relations
Cassie Ercanbrack (Boston)
Media_relations@takeda.com
Tsuyoshi Tada (Tokyo)
Toiawase_kouhou@takeda.co.jp
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