FDA Grants Crinetics Rare Pediatric Disease Designation for Hyperinsulinism Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Crinetics Pharmaceuticals for its experimental therapy for congenital hyperinsulism, a life-threatening condition caused by excess production of insulin.

Hyperinsulinism is a heterogeneous condition in which dangerously low blood sugar levels are caused by increased insulin secretion from pancreatic beta-cells. Congenital hyperinsulism is a severe form of hyperinsulinism driven by genetic mutations in certain genes involved in regulating insulin secretion. The incidence of congenital hyperinsulism is approximately 1 in 30,000 to 50,000 new births in the United States. Early diagnosis is vital to prevent neurological complications due to recurrent low blood sugar, which can result in apnea, seizures, developmental delays, learning disabilities, epilepsy, and even death.

CRN04777 is an investigational, orally available, nonpeptide somatostatin receptor type 5 (SST5) agonist being developed as a treatment for congenital hyperinsulinism.

“We are encouraged by the positive and collaborative interaction we had with the FDA regarding the clinical development of CRN04777 for congenital HI,” said Scott Struthers, founder and CEO. “The receipt of this RPD designation points to the seriousness of the disease for which there is an enormous need for new treatment options. Inspired by the bravery of the patients with congenital HI and their families, we are working diligently to ready CRN04777 for a phase 1 clinical study in early 2021.”

The granting of the designation comes as the program is due to expire at the end of the month. A drug that has been granted RPD designation prior to this date is still eligible to receive the voucher if it receives final FDA approval before September 30, 2022. There is also legislation that has been introduced to extend the program.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes CRN04777 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA. There are no approved therapies for the condition.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

Photo: Scott Struthers, founder and CEO

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.