Crinecerfont approved to treat congenital adrenal hyperplasia
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Key takeaways:
- The FDA approved crinecerfont to treat patients with congenital adrenal hyperplasia.
- The drug directly reduces excess adrenocorticotropic hormone and downstream adrenal androgen production.
Neurocrine Biosciences announced that the FDA has approved crinecerfont as an adjunct to glucocorticoid replacement to control androgens for patients with congenital adrenal hyperplasia.
Crinecerfont (Crenessity), a selective corticotropin-releasing factor type 1 receptor, is the first treatment for congenital adrenal hyperplasia (CAH) that directly reduces excess adrenocorticotropic hormone and downstream adrenal androgen production, according to a press release issued by the company.
As Healio previously reported, data from two trials showed crinecerfont reduced the need for glucocorticoids compared with placebo among adults with CAH and was tied to greater reductions in androstenedione levels vs. placebo for children with the disorder.
“For the past 70 years, glucocorticoids have been used to correct the endogenous cortisol deficiency present in congenital adrenal hyperplasia. In addition, the current treatment paradigm relies on the negative feedback from these exogenous glucocorticoids to lower the overproduction of ACTH and adrenal androgens,” Richard J. Auchus, MD, PhD, professor of pharmacology and internal medicine in the division of metabolism, endocrinology and diabetes at the University of Michigan, told Healio. “To accomplish the latter goal, most patients require higher than physiologic glucocorticoid therapy, including undesirable evening doses. Chronic excessive glucocorticoid exposure has led to many prevalent short- and long-term comorbidities in this population, including growth suppression in children, followed by obesity, hypertension, insulin resistance, mood disorders and osteoporosis. As demonstrated in the clinical trials, CRENESSITY reduces excess ACTH and downstream adrenal androgen production, allowing for glucocorticoid dose reduction and redistribution closer to the normal circadian rhythm. This ‘block-and-replace’ strategy using a CRF-1 antagonist and physiologic glucocorticoid dosing might decrease the known complications associated with chronic exposure to high glucocorticoid doses.”
The medication, which is available in capsules and in an oral solution, will be provided by a specialty pharmacy, PANTHERx Rare, according to the release.
The company also stated in the release that is has launched Neurocrine Access Support, an assistance program for patients, caregivers and health care professionals, which includes a care coordinator to help navigate the insurance process and investigate financial assistance options. Most patients will pay $10 per month or less for the medication, according to the release.
For more information:
Richard J. Auchus, MD, PhD, can be reached at rauchus@med.umich.edu.
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