SAN DIEGO, Oct. 23, 2017 /PRNewswire/ -- Neurocrine
Biosciences, Inc. (NASDAQ: NBIX) today announced that valbenazine,
a novel selective vesicular monoamine transporter 2 (VMAT2), has
been granted Orphan Drug Designation by the U.S. Food and Drug
Administration (FDA) for the treatment of pediatric patients with
Tourette syndrome. Tourette syndrome is a neurological disorder
that becomes evident in early childhood or adolescence and is
characterized by motor and vocal tics. Valbenazine is marketed in
the United States as
INGREZZA® (valbenazine) capsules for the treatment
of adults with tardive dyskinesia.
"We are pleased that the FDA has granted valbenazine orphan drug
designation to treat pediatric patients with Tourette syndrome as
it represents another significant regulatory milestone for our
company," said Malcolm Lloyd-Smith,
Chief Regulatory Officer at Neurocrine Biosciences. "We will
continue our clinical development program for Tourette's focused on
pediatric patients as the first symptoms of Tourette syndrome
typically present early in childhood and represent a significant
challenge for these young patients."
Orphan drug designation is granted by the FDA to drugs that
are intended to treat rare diseases or conditions for patients
in the U.S. The orphan drug designation allows
the orphan drug indication for the drug to be
eligible for a seven-year period of U.S. marketing exclusivity upon
approval of the drug, as well as other development assistance and
financial incentives.
About Tourette Syndrome
Tourette syndrome is a
neurological disorder that consists of rapid, non-rhythmic
stereotyped motor and vocal tics. Motor tics are typically
characterized by facial grimacing, head jerks, extremity movements
and other stereotyped movements. Vocal tics typically include
grunting, throat clearing, and repeating words and phrases. The
average age of onset for Tourette syndrome is six years, with
symptoms reaching their peak severity at approximately age ten.
Tourette syndrome is more commonly diagnosed in males than females
and may be associated with attention deficit hyperactivity disorder
and obsessive-compulsive disorder.
About INGREZZA® (valbenazine) capsules
INGREZZA, a selective VMAT2 inhibitor, is the first FDA approved
product indicated for the treatment of adults with tardive
dyskinesia, a condition associated with uncontrollable, abnormal
and repetitive movements of the trunk, extremities and/or face.
INGREZZA is thought to work by reducing the amount of dopamine
released in a region of the brain that controls movement and motor
function, helping to regulate nerve signaling in adults with
tardive dyskinesia. VMAT2 is a protein in the brain that packages
neurotransmitters, such as dopamine, for transport and release in
presynaptic neurons. INGREZZA, developed in Neurocrine's
laboratories, is novel in that it selectively inhibits VMAT2 with
no appreciable binding affinity for VMAT1, dopaminergic (including
D2), serotonergic, adrenergic, histaminergic, or muscarinic
receptors. Additionally, INGREZZA can be taken as one capsule,
once-daily, together with psychiatric medications such as
antipsychotics or antidepressants. INGREZZA is currently in
clinical development for the treatment of Tourette syndrome.
Important Safety Information
Warnings &
Precautions
Somnolence
INGREZZA can cause
somnolence. Patients should not perform activities requiring
mental alertness such as operating a motor vehicle or operating
hazardous machinery until they know how they will be affected by
INGREZZA.
QT Prolongation
INGREZZA may prolong the QT interval,
although the degree of QT prolongation is not clinically
significant at concentrations expected with recommended dosing.
INGREZZA should be avoided in patients with congenital long QT
syndrome or with arrhythmias associated with a prolonged QT
interval. For patients at increased risk of a prolonged QT
interval, assess the QT interval before increasing the dosage.
Adverse Reactions
The most common adverse reaction
(≥5% and twice the rate of placebo) is somnolence. Other
adverse reactions (≥2% and >placebo) include: anticholinergic
effects, balance disorders/falls, headache, akathisia, vomiting,
nausea, and arthralgia.
You are encouraged to report negative side effects of
prescription drugs to the FDA. Visit MedWatch at
www.fda.gov/medwatch or call 1-800-FDA-1088.
Please see INGREZZA full Prescribing Information at
www.INGREZZA.com/HCP
About Neurocrine Biosciences
Neurocrine
Biosciences is a San Diego based biotechnology
company focused on neurologic, psychiatric and endocrine related
disorders. The Company markets INGREZZA® (valbenazine) capsules
in the United States for the treatment of adults with
tardive dyskinesia. INGREZZA is a novel, selective vesicular
monoamine transporter 2 (VMAT2) inhibitor, and is the first FDA
approved product indicated for the treatment of adults with tardive
dyskinesia. The Company's three late-stage clinical programs
are: elagolix, a gonadotropin-releasing hormone antagonist for
women's health that is partnered with AbbVie Inc.; opicapone,
a novel, once-daily, peripherally-acting, highly-selective
catechol-o-methyltransferase inhibitor under investigation as
adjunct therapy to levodopa in Parkinson's patients; and INGREZZA,
a novel, once-daily, selective VMAT2 inhibitor under investigation
for the treatment of Tourette syndrome.
Neurocrine Biosciences, Inc. news releases are available through
the Company's website via the internet at
http://www.neurocrine.com.
Forward Looking Statements
In addition to
historical facts, this press release contains forward-looking
statements that involve a number of risks and uncertainties. These
statements include, but are not limited to, statements related to
the benefits to be derived from INGREZZA and whether results from
INGREZZA's clinical trials are indicative of real-world
results. Among the factors that could cause actual results to
differ materially from those indicated in the forward-looking
statements are: risks and uncertainties associated with
Neurocrine's business and finances in general; risks that the
Company may not complete its development program in Tourette
syndrome timely, or at all; risks and uncertainties relating to
competitive products and technological changes that may limit
demand for INGREZZA; risks associated with the Company's dependence
on third parties for development and manufacturing activities
related to INGREZZA and the ability of the Company to manage these
third parties; risks that the FDA or other regulatory authorities
may make adverse decisions regarding INGREZZA; risks that INGREZZA
clinical trials may not be predictive of real-world results or of
results in subsequent clinical trials; risks that INGREZZA may be
alleged to infringe upon the proprietary rights of third parties,
or have unintended side effects, adverse reactions or
incidents of misuse; and other risks described in the Company's
periodic reports filed with the Securities and Exchange Commission,
including without limitation the Company's Quarterly Report on Form
10-Q for the quarter ended June 30,
2017. The Company disclaims any obligation to update the
statements contained in this press release after the date
hereof.
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SOURCE Neurocrine Biosciences, Inc.