FDA Grants Rare Pediatric Disease Designation to OR-440 for Treatment of Adrenocortical Carcinoma


OR-449 is being developed for both adult and pediatric patients with adrenocortical carcinoma, as well as other cancers known to express a high level of steroidogenic factor-1.

Officials with the FDA have granted Rare Pediatric Disease Designation (RPDD) to OR-440, an investigational drug for the treatment of pediatric adrenocortical carcinoma (ACC).

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

“We are gratified that OR-449 has received an RPDD from the FDA,” said Scott Thacher, PhD, CEO of Orphagen, in a press release. “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.”

ACC is a rare and aggressive cancer of the adrenal gland. If the tumor has not metastasized, surgical removal can be an effective treatment. However, ACC tumors become metastatic for most patients, making them difficult to control and with 5-year survival rates between 10% and 20% in both adults and children.

In clinical practice, SF-1 is widely used as a marker for ACC and is a potential therapeutic target for both adult and pediatric patients with ACC. It is commonly amplified at the gene level in pediatric patients with ACC and is recognized as a cell lineage marker in the FDA’s Pediatric Cancer Target List.

Under the RPDD, the FDA defines a rare pediatric disease as a serious or life-threatening disease primarily affecting individuals aged from birth to 18 years and affecting fewer than 200,000 patients in the United States. To address drug development challenges in these unique populations, Congress has reauthorized the Creating Hope Act, under which companies can receive a Priority Review Voucher following approval of a product with an RPDD. Companies are eligible for this if the marketing application receives FDA approval by September 30, 2026, under current legislation, or at a later date if the act is extended by Congress.

“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,” Thacher said in the press release.


Orphagen Pharmaceuticals Receives Rare Pediatric Disease Designation From FDA for OR-449 for the Treatment of Pediatric Adrenocortical Carcinoma. News release. Orphagen Pharmaceuticals; January 17, 2023. Accessed January 19, 2023. https://www.orphagen.com/orphagen-pediatric-adrenocortical-carcinoma/

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