SILVER
SPRING, Md., Dec. 13,
2024 /PRNewswire/ -- Today, the U.S. Food and Drug
Administration approved Crenessity (crinecerfont) to be used
together with glucocorticoids (steroids) to control androgen (a
testosterone-like hormone) levels in adults and pediatric patients
4 years of age and older with classic congenital adrenal
hyperplasia (CAH).
"Today's approval provides an important advance for patients
with classic congenital adrenal hyperplasia and highlights the
FDA's continued commitment to advancing effective and safe
treatments for rare diseases," said Theresa
Kehoe M.D., director of the Division of General
Endocrinology in the FDA's Center for Drug Evaluation and
Research. "The FDA will continue working with patients, drug
companies and health care providers to address the unmet medical
needs of the rare disease community."
Classic congenital adrenal hyperplasia is a rare genetic
condition affecting the adrenal glands, which produce hormones such
as cortisol and androgens. Patients with classic CAH do not produce
enough cortisol and produce too many androgens. These patients
require high doses of glucocorticoids (more than is typically
needed to replace the deficient cortisol) because the
glucocorticoids also help to reduce the excess levels of androgens.
Crenessity works by reducing excessive adrenal androgen production,
which helps reduce the amount of glucocorticoid treatment
needed.
Crenessity's approval is based on two randomized, double-blind,
placebo-controlled trials in 182 adults and 103 children with
classic CAH. In the first trial, 122 adults received Crenessity
twice daily and 60 received placebo twice daily for 24 weeks. After
the first four weeks of the trial, the glucocorticoid dose was
reduced to replacement levels, then adjusted based on levels of
androstenedione, an androgen hormone. The primary measure of
efficacy was the change from baseline in the total glucocorticoid
daily dose while maintaining androstenedione control at the end of
the trial. The group that received Crenessity reduced their daily
glucocorticoid dose by 27% while maintaining control of
androstenedione levels, compared to a 10% daily glucocorticoid dose
reduction in the group that received placebo.
In the second trial, 69 pediatric patients received Crenessity
twice daily and 34 received placebo twice daily for 28 weeks. The
primary measure of efficacy was the change from baseline in serum
androstenedione at week 4. The group that received Crenessity
experienced a statistically significant reduction from baseline in
serum androstenedione, compared to an average increase from
baseline in the placebo group. At the end of the trial, patients
assigned to Crenessity were able to reduce their daily
glucocorticoid dose by 18% while maintaining control of
androstenedione levels compared to an almost 6% daily
glucocorticoid dose increase in patients assigned to placebo.
Crenessity has a warning for acute adrenal insufficiency or
adrenal crisis, which can occur if patients with underlying adrenal
insufficiency who do not also receive adequate doses of
glucocorticoid replacement therapy in situations associated with
increased cortisol need (i.e., "stress dose steroids"). Crenessity
should not be taken by patients with hypersensitivity to
Crenessity's active ingredient or any of its components.
Because Crenessity is metabolized by a specific enzyme,
using Crenessity at the same time as drugs that activate that
enzyme can decrease the amount of Crenessity exposure and reduce
Crenessity's efficacy. Information about recommended dose
adjustments is included in Crenessity's full prescribing
information.
The most common side effects of Crenessity in adults include
fatigue, dizziness and arthralgia (joint pain). For pediatric
patients, the most common side effects are headache, abdominal pain
and fatigue.
The FDA granted Crenessity Fast Track, Breakthrough Therapy,
Orphan Drug and Priority Review designations.
The FDA granted the approval of Crenessity to Neurocrine
Biosciences, Inc.
Media Contact: FDA Office of Media Affairs,
301-796-4540
Consumer Inquiries: Email or 888-INFO-FDA
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of Health and Human Services, protects the public health by
assuring the safety, effectiveness, and security of human and
veterinary drugs, vaccines and other biological products for human
use, and medical devices. The agency also is responsible for the
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regulating tobacco products.
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SOURCE U.S. Food and Drug Administration