According to the recent announcement by the US FDA, Crenessity (crinecerfont) developed by Neurocrine Biosciences has been approved for marketing as an adjuvant therapy in combination with glucocorticoids for the treatment of classic congenital adrenal hyperplasia (CAH) in adults and pediatric patients aged 4 and above.

 

 

 

Data shows that Crinecerfont is an oral, selective corticotropin releasing factor 1 receptor (CRF1) antagonist that reduces and controls excess adrenal androgens through a hormone independent mechanism, used to treat congenital adrenal gland hyperplasia caused by 21 hydroxylase deficiency.

 

 

 

Research has shown that antagonizing CRF1 in the pituitary gland can lower levels of adrenocorticotropic hormone, thereby reducing the production of adrenal androgens and potentially reducing symptoms associated with classical CAH. Crinecerfont has received breakthrough therapy recognition, orphan drug qualification, and priority review qualification from the US FDA.

 

 

 

This is a treatment method specifically designed for typical congenital adrenal hyperplasia, which can directly reduce the production of excessive adrenocorticotropic hormone and downstream adrenal androgen, thereby reducing the dosage of glucocorticoids. According to relevant sources, the effect of the drug is to reduce the excessive production of adrenal androgens, thereby reducing the required amount of glucocorticoid therapy, marking a significant shift in treatment methods.

 

 

 

It is reported that this approval is based on the results of placebo-controlled trials involving both adults and children. According to the previously published Phase 3 CAHtalyst pediatric study data, compared to placebo, treatment with crinecerfont resulted in a statistically significant decrease in serum androstenedione levels in patients at week 4 compared to baseline (p=0.0002). This result is consistent with the results of the Phase 3 CAHtalyst Adult Study, indicating that crinecerfont has shown efficacy in patients of different age groups. At week 28, compared to placebo, treatment with crinecerfont reduced the daily required dose of glucocorticoids while maintaining androgen control in patients (p<0.0001). In addition, Crinecerfont has demonstrated good safety and tolerability in its research.

 

 

 

Neurocrine previously stated in a press release that if approved, this drug would be the first new treatment option for CAH in 70 years.

 

 

 

The industry said that rare diseases, due to their very low incidence rate, limited number of patients, and high drug research and development costs, were "forgotten corners" in the medical field for a long time.

 

 

 

It is reported that in the field of rare diseases, China is accelerating the construction of a road to ensure the prevention and treatment of rare diseases through the efforts of multiple departments, building a bridge of life for patients, and continuously achieving breakthroughs in the accessibility and affordability of medication. In recent years, drugs for the treatment of rare diseases such as spinal muscular atrophy (SMA), Gaucher's disease, and myasthenia gravis have been included in the list... Since the establishment of the National Medical Insurance Administration in 2018, the list of medical insurance drugs has been adjusted for seven consecutive years. At present, more than 90 rare disease drugs have been included in the national medical insurance drug catalog, and the level of rare disease drug protection has steadily improved.