In a recent press release, clinical-stage pharmaceutical company Crinetics Pharmaceuticals announced that their investigational drug candidate, CRN04777, received Rare Pediatric Disease designation from the FDA. The therapy is designed to treat patients with congenital hyperinsulinism, a condition characterized by the over secretion of insulin. Overall, the therapy aligns with Crinetics’ goal of treating patients with endocrine disease and endocrine-related tumors.
Rare Pediatric Disease Designation
Developed by Crinetics Pharmaceuticals, CRN04777 is an investigational, orally administered nonpetide somatostatin receptor type 5 (SST5) agonist. In medicine, an agonist mimics and activates certain receptors. In this case, it activates and mimics SST5. Somatostatin inhibits the release of certain secretions and hormones. As a result, CRN04777 prevents the overrelease of insulin, thus reducing symptoms and improving health.
The priority review voucher program for rare pediatric disease drugs provides an additional benefit by shortening the FDA drug submission review process from the standard ten months to six; however, the priority review process does not require approval within this timeframe.
According to the current statutory sunset provisions for the Rare Pediatric Disease Priority Review Voucher Program, after September 30, 2020, FDA may only award a voucher for an approved rare pediatric disease product application if the sponsor has rare pediatric disease designation for the drug, and that designation was granted by September 30, 2020. After September 30, 2022, FDA may not award any rare pediatric disease priority review vouchers.
There are eight forms of congenital hyperinsulinism, a rare condition caused by excess insulin secretion. Each form is caused by something different, ranging from genetics to premature birth. Excess insulin causes low blood sugar, which can cause neurological problems. An estimated 1 in 50,000 babies has congenital hyperinsulinism.
- Feeding difficulties
- Extreme hunger
- Muscle weakness and shakiness
- Extreme fatigue
- Rapid heart rate
Without treatment, congenital hyperinsulinism can also cause a variety of serious complications. These include comas, seizures, intellectual delays, vision loss, and difficulty breathing (apnea). In some cases, this condition is fatal. Learn more about congenital hyperinsulinism here.