Dina Matos, Executive Director of the CARES Foundation, discusses the recent approval of crinecerfont to treat patients with congenital adrenal hyperplasia (CAH) by the U.S. Food and Drug Administration (FDA).
CAH refers to a group of genetic conditions that affect the adrenal glands. It is caused by a deficiency of an enzyme needed by the adrenal glands to make these hormones and often overproduce androgens. For example, females with a severe form of the condition may have ambiguous genitalia at birth and if not properly diagnosed, develop dehydration, poor feeding, diarrhea, vomiting and other health problems soon after. People with milder forms may not be diagnosed with the condition until adolescence or adulthood when they experience early signs of puberty or fertility problems.
The FDA has recently approved crinecerfont capsules and oral solution for glucocorticoid replacement treatment in patients with classic CAH. Crinecerfont is a potent and selective oral corticotropin-releasing factor type 1 receptor antagonist. It is the first and only classic CAH treatment that directly reduces excess ACTH and downstream adrenal androgen production.
The FDA approval follows results from the CAHtalyst pediatric and adult phase 3 global registrational studies. In both studies, crinecerfont was observed to enable lower steroid doses and decreased androgen levels. Both studies met their primary endpoints. Additionally, crinecerfont was well-tolerated with few adverse effects.
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To learn more about CAH and other rare endocrine disorders, visit https://checkrare.com/diseases/endocrine-disorders/
