- CRENESSITY, a First-in-Class Treatment for Classic Congenital
Adrenal Hyperplasia (CAH), is now commercially available in the
U.S.
- CAH-trained pharmacists are available 24/7 to support patients
with prescriptions exclusively through PANTHERx Rare, a specialty
pharmacy
- Neurocrine Access Support is available to provide free,
comprehensive access and support information to patients,
caregivers and healthcare providers
SAN
DIEGO, Dec. 20, 2024 /PRNewswire/
-- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today
announced CRENESSITY™ (crinecerfont) is now commercially available
in the United States. CRENESSITY
was recently approved by the U.S. Food and Drug Administration
as an adjunctive treatment to glucocorticoid replacement to
control androgens in adult and pediatric patients four years of age
and older with classic congenital adrenal hyperplasia (CAH).
CRENESSITY, a potent and selective oral corticotropin-releasing
factor type 1 receptor (CRF1) antagonist, is a
first-in-class therapy for classic CAH that directly reduces
adrenocorticotropic hormone and downstream adrenal androgen
production. It is the first and only classic CAH treatment
that allows people to take lower doses of glucocorticoids while
maintaining or improving their androgen levels.
"Individuals with CAH and their families have faced ongoing
challenges with managing the condition with high-dose steroids
alone for the past 70 years," said Kyle W.
Gano, Ph.D., Chief Executive Officer, Neurocrine
Biosciences. "We're proud to now provide CRENESSITY to the
community, and we are committed to supporting patients in obtaining
treatment with CRENESSITY through our comprehensive assistance
program."
CRENESSITY is exclusively available through PANTHERx Rare, a
specialty pharmacy, to centralize and simplify CRENESSITY
prescriptions. PANTHERx Rare has CAH-trained pharmacists available
24/7 to patients, caregivers and healthcare providers to answer
questions and address concerns.
Neurocrine Biosciences is committed to supporting patients
in obtaining treatment with CRENESSITY by offering Neurocrine
Access Support, a free, comprehensive assistance program created
for patients, caregivers and healthcare providers. It offers a
range of options specifically designed to ensure patients with CAH
have everything they need to begin and continue taking CRENESSITY.
A dedicated Care Coordinator, backed by a team, is available to
help patients and caregivers navigate the insurance process and
identify appropriate financial assistance options. The company
expects 90% of patients will have a monthly copay of $12 or less. For more information, visit
www.nbiaccess.com/crenessity or call 1-855-CRNSITY (276-7489)
Monday-Friday 8 am-8 pm ET.
The U.S. Food and Drug Administration approval of CRENESSITY was
supported by the largest-ever clinical trial program of classic
CAH, the CAHtalyst™ Pediatric study, conducted in
ages four to 17, and the CAHtalyst Adult study.
View the press release announcing the initial FDA approval of
CRENESSITY here.
For more information about CRENESSITY, visit Crenessity.com.
About Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) is a rare genetic condition
that results in an enzyme deficiency that alters the production of
adrenal steroid hormones, such as cortisol, aldosterone and adrenal
androgens, which are essential for life. Approximately 95% of CAH
cases are caused by variants of the CYP21A2 gene that leads to
deficiency of the enzyme 21-hydroxylase (21-OH). Severe deficiency
of this enzyme leads to an inability of the adrenal glands to
produce enough cortisol and, in approximately 75% of cases,
aldosterone. Because individuals with CAH are still able to produce
androgens, the unused precursors that would normally be used to
make cortisol instead result in the production of excess amounts of
androgens. If left untreated, CAH can result in salt wasting,
dehydration and even death.
Historically, exogenous glucocorticoids (GCs) have been used not
only to correct the endogenous cortisol deficiency, but doses used
are higher than cortisol replacement needed (supraphysiologic) to
lower the levels of adrenocorticotropic hormone (ACTH) and adrenal
androgens. However, GC treatment at high doses has been associated
with serious and significant complications of steroid excess,
including metabolic issues such as weight gain and diabetes,
cardiovascular disease and osteoporosis. Additionally, long-term
treatment with high-dose GCs may have psychological and cognitive
impact, such as changes in mood and memory. Adrenal androgen excess
has been associated with abnormal bone growth and development in
pediatric patients, female health problems such as excess facial
hair growth and menstrual irregularities, testicular rest tumors in
males and fertility issues in both sexes.
About The CAHtalyst™ Studies
The Phase 3
CAHtalyst™ global registrational studies were
designed to evaluate the safety, efficacy and tolerability of
CRENESSITY in children and adults with classic congenital adrenal
hyperplasia (CAH) due to 21-hydroxylase deficiency. The CAHtalyst
studies were the largest-ever clinical trial program in classic
CAH, including 285 pediatric and adult patients.
The CAHtalyst Pediatric study included 103 pediatric patients
aged four to 17 years. The study tested two questions. The first
question evaluated whether four weeks of CRENESSITY treatment could
improve androgen control. The second question evaluated whether an
additional 24 weeks of CRENESSITY treatment enabled customized
glucocorticoid (GC) down-titration while androstenedione levels
were maintained or improved. The CAHtalyst Adult study included 182
adult patients aged 18 to 58 years. Similarly, the first question
of the study evaluated whether four weeks of CRENESSITY treatment
could improve androgen control, and the second question evaluated
whether an additional 20 weeks of CRENESSITY treatment enabled GC
reduction to physiologic range while androstenedione levels were
maintained or improved.
Data from the CAHtalyst Phase 3 studies supported approval of
CRENESSITY by the U.S. Food and Drug Administration in December 2024. The open-label extension treatment
portions of both studies are ongoing.
About CRENESSITY™ (crinecerfont)
CRENESSITY™ is a
potent and selective, oral corticotropin-releasing factor type 1
receptor (CRF1) antagonist developed to reduce and
control excess adrenocorticotropic hormone (ACTH) and adrenal
androgens through a non-glucocorticoid (GC) mechanism for the
treatment of classic congenital adrenal hyperplasia (CAH).
Antagonism of CRF1 receptors in the pituitary has
been shown to decrease ACTH levels, which in turn decreases the
production of adrenal androgens and potentially the symptoms
associated with CAH. The robust clinical study data demonstrate
that lowering adrenal androgen levels with CRENESSITY enables
lower, more physiologic dosing of GCs to replace missing
cortisol.
CRENESSITY comes in capsules and an oral solution. The capsule
formulation is available in 50 mg and 100 mg doses. The oral
solution is available as a 50 mg/mL strength formulation. For
adults 18 years and older, the recommended dosage is 100 mg twice
daily taken orally with a meal. For pediatric patients four to 17
years of age weighing less than 55 kg (121 lbs), the recommended
dosage is based on body weight and is administered twice daily,
taken orally with a meal. For pediatric patients weighing more than
55 kg (121 lbs), the recommended dosage is 100 mg twice daily taken
orally with a meal. Healthcare providers can work with patients to
determine the appropriate formulation for use depending on patient
needs. Patients receiving CRENESSITY should continue GC therapy for
cortisol replacement.
Important Information
Approved Uses
CRENESSITY (crinecerfont) is a
prescription medicine used together with glucocorticoids (steroids)
to control androgen (testosterone-like hormone) levels in adults
and children 4 years of age and older with classic congenital
adrenal hyperplasia (CAH).
IMPORTANT SAFETY INFORMATION
Do not take CRENESSITY if you:
Are allergic to crinecerfont, or any of the ingredients in
CRENESSITY.
CRENESSITY may cause serious side effects,
including:
Allergic Reactions. Symptoms of an allergic reaction
include tightness of the throat, trouble breathing or swallowing,
swelling of the lips, tongue, or face, and rash. If you have an
allergic reaction to CRENESSITY, get emergency medical help right
away and stop taking CRENESSITY.
Risk of Sudden Adrenal Insufficiency or Adrenal
Crisis With Too Little Glucocorticoid (Steroid)
Medicine. Sudden adrenal insufficiency or adrenal crisis can
happen in people with congenital adrenal hyperplasia who are not
taking enough glucocorticoid (steroid) medicine. You should
continue taking your glucocorticoid (steroid) medicine during
treatment with CRENESSITY. Certain conditions such as infection,
severe injury, or shock may increase your risk for sudden adrenal
insufficiency or adrenal crisis. Tell your healthcare provider if
you get a severe injury, infection, illness, or have planned
surgery during treatment. Your healthcare provider may need to
change your dose of glucocorticoid (steroid) medicine.
Before taking CRENESSITY, tell your healthcare provider about
all of your medical conditions, including if you are pregnant
or plan to become pregnant, or are breastfeeding or plan to
breastfeed.
Tell your healthcare provider about all the medicines you
take, including prescription and over-the counter medicines,
vitamins, and herbal supplements.
The most common side effects of CRENESSITY in adults
include tiredness, headache, dizziness, joint pain, back
pain, decreased appetite, and muscle pain.
The most common side effects of CRENESSITY in children
include headache, stomach pain, tiredness, nasal
congestion, and nose bleeds.
These are not all the possible side effects of CRENESSITY. Call
your healthcare provider for medical advice about side effects. 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.
Dosage Forms and Strengths: CRENESSITY is available in 50
mg and 100 mg capsules and as an oral solution of 50 mg/mL.
Please see full Prescribing Information.
About Neurocrine Biosciences, Inc.
Neurocrine Biosciences is a leading neuroscience-focused,
biopharmaceutical company with a simple purpose: to relieve
suffering for people with great needs, but few options. We are
dedicated to discovering and developing life-changing treatments
for patients with under-addressed neurological, neuroendocrine and
neuropsychiatric disorders. The company's diverse portfolio
includes FDA-approved treatments for tardive dyskinesia, chorea
associated with Huntington's disease, classic congenital adrenal
hyperplasia, endometriosis* and uterine fibroids,* as well as a
robust pipeline including multiple compounds in mid- to late-phase
clinical development across our core therapeutic areas. For three
decades, we have applied our unique insight into neuroscience and
the interconnections between brain and body systems to treat
complex conditions. We relentlessly pursue medicines to ease the
burden of debilitating diseases and disorders, because you deserve
brave science. For more information,
visit neurocrine.com, and follow the company
on LinkedIn, X (formerly Twitter) and Facebook.
(*in collaboration with AbbVie)
The NEUROCRINE BIOSCIENCES Logo Lockup and YOU DESERVE BRAVE
SCIENCE are registered trademarks of Neurocrine Biosciences,
Inc. CRENESSITY and CAHtalyst are trademarks
of Neurocrine Biosciences, Inc.
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 regarding the potential
benefits to be derived from CRENESSITY for the treatment of
classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase
deficiency; the value and benefits CRENESSITY brings to patients
with CAH; the ability of Neurocrine Biosciences to ensure patients
have access to CRENESSITY; and whether the results from our
clinical trials of CRENESSITY are indicative of real-world results.
Factors that could cause actual results to differ materially from
those stated or implied in the forward-looking statements include,
but are not limited to, the following: risks and uncertainties
associated with Neurocrine Biosciences' business and finances in
general, as well as risks and uncertainties associated with the
commercialization of CRENESSITY; whether CRENESSITY receives
adequate reimbursement from third-party payors; the degree and pace
of market uptake of CRENESSITY; risks and uncertainties relating to
competitive products and technological changes that may limit
demand for CRENESSITY; risks associated with the Company's
dependence on third parties for development and manufacturing
activities related to CRENESSITY, and the ability of the Company to
manage these third parties; risks that additional regulatory
submissions for CRENESSITY or other product candidates may not
occur or be submitted in a timely manner; risks that the FDA or
other regulatory authorities may make adverse decisions regarding
CRENESSITY; risks that post-approval CRENESSITY commitments or
requirements may be delayed; risks that CRENESSITY may be precluded
from commercialization by the proprietary or regulatory rights of
third parties, or have unintended side effects, adverse reactions
or incidents of misuse; risks and uncertainties relating to
competitive products and technological changes that may limit
demand for CRENESSITY; 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 September 30,
2024. Neurocrine Biosciences disclaims any obligation to
update the statements contained in this press release after the
date hereof other than required by law.
© 2024 Neurocrine Biosciences, Inc. All Rights Reserved.
CP-CFT-US-0294 12/2024
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SOURCE Neurocrine Biosciences, Inc.