RARE Daily

FDA Grants Rare Pediatric Disease Designation for Orphagen’s OR-449 for Pediatric Adrenocortical Carcinoma

January 17, 2023

The U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation for to Orphagen Pharmaceuticals for OR-449 for the treatment of pediatric adrenocortical carcinoma.

Photo: Scott Thacher, CEO of Orphagen

Adrenocortical carcinoma (ACC) is a rare and aggressive cancer of the adrenal gland. Surgical removal of an affected adrenal gland is effective treatment if the tumor has not metastasized. Once an ACC tumor becomes metastatic, as it does for most patients, it is difficult to control and five-year survival in both adult and pediatric patients with metastatic disease is low, about 10 to 20 percent. The estimated annual incidence of ACC in the United States is 600 patients/year. In clinical practice, SF-1 is widely used as a marker for ACC, and it is recognized as a potential therapeutic target for both adult and pediatric ACC. SF-1 is commonly amplified at the gene level in pediatric ACC, and SF-1 is recognized as a cell lineage marker in the FDA’s Pediatric Cancer Target List.

OR-449 is a selective, first-in-class, potent and orally bioavailable small molecule antagonist to steroidogenic factor-1 (SF-1 or NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland. Orphagen is developing OR-449 for both the adult and pediatric forms of ACC as well as other cancers known to express a high level of SF-1.

“We currently plan to file an Investigational New Drug application with the FDA later this year to support initiation of a phase 1 clinical trial,” said Scott Thacher, CEO of Orphagen. “We speak to clinicians regularly who remind us of the urgent need for an improved therapy for patients with ACC, and this drives our commitment to the clinical development of OR-449, our first internal program to reach this stage.”

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may be eligible for a voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product or sold to another sponsor for priority review of their marketing application.

Recently, Bluebird Bio sold two priority review vouchers: one to Argenx for $102 million and one to Bristol Myers Squibb for $95 million.

Author: Rare Daily Staff

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