UK’s MHRA Grants Crinetics Innovation Passport for Hyperinsulinism Therapy

The U.K. Medicines and Healthcare products Regulatory Agency has granted Crinetics Pharmaceuticals CRN04777 an Innovation Passport for the treatment of the rare and life-threatening condition congenital hyperinsulinism.

Photo: Scott Struthers, president and CEO of Crinetics

The designation gives Crinetics access to the Innovative Licensing and Access Pathway. The pathway was launched in the United Kingdom in 2021 with the goal of reducing the time to market for innovative medicines that treat life-threatening or seriously debilitating conditions and conditions for which there is a significant unmet patient need. It aims to achieve this goal by enabling enhanced coordination between sponsors and MHRA leading up to Marketing Authorization Application submissions and by providing the opportunity for accelerated reviews.

Congenital hyperinsulism (HI) afflicts infants and young children and can lead to complications such as seizures, coma, and brain damage. Many children with the disease require intensive 24-hour glucose management, which is burdensome and provides limited efficacy.

CRN04777 is a highly optimized, orally available, small molecule somatostatin receptor type 5  selective agonist that is designed to reduce the excess secretion of insulin in patients with congenital monogenic and syndromic HI, and other conditions of excess insulin. Oral administration of CRN04777 has been shown to potently inhibit insulin secretion and normalize glucose levels in preclinical models of hyperinsulinism.

A phase 1 clinical study was conducted in healthy volunteers to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of CRN04777, the results of which demonstrated pharmacologic proof-of-concept for CRN04777, with strong dose-dependent suppression of insulin secretion.

“We believe the Innovation Passport provides important external validation for the CRN04777 program and shows that the MHRA recognizes the struggle congenital HI patients and caregivers face each day,” Scott Struthers, president and CEO of Crinetics.

The U.S. Food and Drug Administration granted rare pediatric disease designation for CRN04777 for the treatment of congenital hyperinsulinism. A rare pediatric disease is defined as a serious or life-threatening disease, which primarily affects individuals aged from birth to 18 years and affects fewer than 200,000 people in the United States.