CRN04777 Given Rare Pediatric Disease Designation for Congenital Hyperinsulinism


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 FDA granted Rare Pediatric Disease designation to CRN04777. This is granted to therapies designed to treat pediatric patients (0-18 years) with rare diseases. In this case, rare diseases are defined as those which affect under 200,000 American patients. Alongside this designation, drug developers are also given priority review vouchers.
How do these vouchers work? According to Premier Research:
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.
However, the FDA is soon ending this program. On their website, the FDA explains:
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.
In the interim, Crinetics hopes to hold the first Phase 1 clinical trial for CRN04777 by early 2021.

Congenital Hyperinsulinism

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.

Symptoms include:

  • Feeding difficulties
  • Extreme hunger
  • Irritability
  • Lethargy
  • Muscle weakness and shakiness
  • Confusion
  • 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.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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