FDA Grants Breakthrough Therapy Designation for Oral Congenital Adrenal Hyperplasia Drug

Posted on January 5, 2024 by MaryO

Key takeaways:

  • Crinecerfont was granted FDA breakthrough therapy designation for the treatment of congenital adrenal hyperplasia.
  • The medication met primary and secondary endpoints in a pair of phase 3 trials.

The FDA granted breakthrough therapy designation for an oral non-glucocorticoid medication for the treatment of congenital adrenal hyperplasia, according to an industry press release.

Crinecerfont (Neurocrine Biosciences) is a selective corticotropin-releasing factor type 1 receptor antagonist under development to lower excess adrenal androgens for people with congenital adrenal hyperplasia due to 21-hyroxylase deficiency.

The medication met its primary and secondary endpoints in two phase 3 CAHtalyst trials, one assessing use of crinecerfont by children and the other by adults. In the pediatric trial, children and adolescents receiving crinecerfont had a decrease in serum androstenedione from baseline to 4 weeks. Participants receiving the medication also had a greater reduction in daily glucocorticoid at 28 weeks than placebo. As Healio previously reported, in the adult trial, crinecerfont was associated with a greater reduction in daily glucocorticoid while maintaining androgen control compared with placebo. The most common adverse events in the pediatric study were headache, fever, vomiting, upper respiratory tract infection and nasopharyngitis. Among adults, the most common adverse events were fatigue, headache and COVID-19 infection. No serious adverse events related to crinecerfont were reported.

Breakthrough therapy is the latest designation granted to crinecerfont by the FDA. The medication was previously granted fast track and rare pediatric disease designations.

“We are very pleased that the FDA granted breakthrough therapy designation for crinecerfont, thus recognizing both the seriousness of congenital adrenal hyperplasia and the significant unmet need currently faced by patients and families living with this condition,” Eiry W. Roberts, MD, Chief Medical Officer for Neurocrine Biosciences, said in a press release. “The outstanding safety and efficacy results from the phase 3 CAHtalyst studies in pediatric and adult patients suggest that crinecerfont has the potential to represent a substantial improvement over current standard of care in congenital adrenal hyperplasia by controlling androgen levels and allowing for reduced steroid doses. We remain on track to submit the new drug application in 2024.”

From https://www.healio.com/news/endocrinology/20231206/fda-grants-breakthrough-therapy-designation-for-oral-congenital-adrenal-hyperplasia-drug?utm_source=selligent&utm_medium=email&utm_campaign=news&fbclid=IwAR2WXDd3ajhKG0s2h0XD9ZQAstUkSotJYl1KLicH3gmxEPF6hvg6sZu2dCU

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Evaluation of Psoriasis Patients With Long-Term Topical Corticosteroids for Their Risk of Developing Adrenal Insufficiency, Cushing’s Syndrome and Osteoporosis

Posted on January 3, 2024 by MaryO

In this study, we will investigate the possible side effects of psoriasis patients using long-term topical corticosteroids (TCS) such as adrenal insufficiency, Cushing’s Syndrome (CS) and osteoporosis and determine how these side effects develop.

Forty-nine patients were included in the study. The patients were divided into two groups based on the potency of the topical steroid they took and the patients’ ACTH, cortisol and bone densitometer values were evaluated.

There was no significant difference between the two groups regarding the development of surrenal insufficiency, CS and osteoporosis. One patient in group 1 and 4 patients in group 2 were evaluated as iatrogenic CS. ACTH stimulation tests of these patients in group 2 showed consistent results with adrenal insufficiency, while no adrenal insufficiency was detected in the patient in Group 1. Patients who used more than 50g of superpotent topical steroids per week compared to patients who used 50g of superpotent topical steroids per week. It was identified that patients who used more than 50g of superpotent topical steroids had significantly lower cortisol levels, with a negatively significant correlation between cortisol level and the amount of topical steroid use ( < .01).Osteoporosis was detected in 3 patients in group 1 and 8 patients in Group 2. Because of the low number of patients between two groups, statistical analysis could not be performed to determine the risk factors.

Our study is the first study that we know of that investigated these three side effects. We have shown that the development of CS, adrenal insufficiency and osteoporosis in patients who use topical steroids for a long time depends on the weekly TCS dosage and the risk increases when it exceeds the threshold of 50 grams per week. therefore, our recommendation would be to avoid long-term use of superpotent steroids and to choose from the medium-potent group if it is to be used.

ABOUT THE CONTRIBUTORS

Betul Erdem

Department of Dermatology, Van Training and Research Hospital, Van, Turkey.

Muzeyyen Gonul

Department of Dermatology, Ministry of Health, Ankara Etlik City Hospital, Ankara, Turkey.

Ilknur Ozturk Unsal

Department of Endocrine and Metabolic Disease, Ministry of Health, Ankara Etlik City Hospital, Ankara, Turkey.

Seyda Ozdemir Sahingoz

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