Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia

According to a story from Healio, the results from a recent phase 2A clinical trial have demonstrated the effectiveness of an experimental drug in treating congenital adrenal hyperplasia (CAH). The investigational product candidate, known as tildacerfont, is being developed by Spruce Biosciences. With no treatments for the disease approved by the US Food and Drug Administration (FDA), the findings could herald a potential breakthrough in the treatment of the disorder.

About Congenital Adrenal Hyperplasia

Congenital adrenal hyperplasia (CAH) is a group of several related genetic disorders that appear as a result of certain genetic mutations that affect the production of sex steroids, mineralocorticoids, and glucocorticoids. The different variants of congenital adrenal hyperplasia are distinguished by which gene is affected. Symptoms vary depending on the sex of the patient and the form of the disorder. These symptoms can include hypogonadism, infertility, vomiting, undervirilization (in males), ambiguous genitalia (in females), early puberty or delayed puberty, rapid growth, menstrual irregularities, and excessive facial hair. Management of congenital adrenal hyperplasia typically involves supplementation of the substance that is affected, such as replacement glucocorticoids, mineralocorticoids, or sex steroids. Other treatments may be used to control growth rate. There is no known cure for these disorders. To learn more about congenital adrenal hyperplasia, click here.

About The Trial

Adult patients in the study were treated once a day with the drug for a period of twelve weeks. The drug was able to significantly reduce androgen levels. 11 patients in total participated in the trial and received tildacerfont at a dose level of 400 mg. The treatment was able to bring down androgen levels by a mean of 74 percent for adrenocorticotropic hormone (ACTH), 55 percent for androstenedione, and 82 percent for 17-hydroxyprogesterone (17-OHP), a steroid hormone.

About Tildacerfont

Tildacerfont is designed as an antagonist of the corticotropin-releasing factor type-1 receptor that allows it to directly target ACTH and other hormones. These findings suggest that it could be the first non-steroidal method for treating the disorder. However, the drug is still far from approval and must continue through the remaining regulatory process. Hopefully, future trials will reinforce the encouraging results from this study.


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