FDA grants rare pediatric disease designation to OR-499 for adrenocortical carcinoma


Orphagen Pharmaceuticals has announced their drug OR-499 received a rare pediatric disease designation for treating pediatric adrenocortical carcinoma from the US Food and Drug Administration.

The US Food and Drug Administration (FDA) has given OR-449 (Orphagen Pharmaceuticals) a rare pediatric disease designation (RPDD) for treating pediatric adrenocortical carcinoma (ACC).

ACC is a form of cancer present in the adrenal gland. Removing the affected gland before a tumor metastasizes is the most effective treatment, but management becomes difficult if a tumor has spread. After this point, 5-year survival is low in adult and pediatric patients, at about 10% to 20%. Tumors metastasize in most patients with ACC.

In the United States, ACC has an estimated incidence of 600 patients annually. Steroidogenic factor-1 (SF-1 or NR5A1) is often used as a marker for ACC in children and adults. 

SF-1 is an orphan nuclear reactor vital for the adrenal gland to develop. OR-449 acts as a small molecule antagonist to SF-1, and ACC is developing it for adult and pediatric ACC, along with other cancers which present with high levels of SF-1.

The Federal Food, Drug, and Cosmetic Act defines a rare pediatric disease as a life-threatening disease mainly present in individuals aged under 18 years, with less than 200,000 people in the United States affected. Companies given RPDD may receive a priority review voucher, which can be redeemed for priority review of the product.

Orphagen Pharmaceuticals is a biotech company developing small molecule ligands for modulating orphans or unexplored members of the nuclear receptor family. The company is exploring lead molecules which may be effective in autoimmune disease and oncology.

According to Scott Thacher, PhD, CEO of Orphagen Pharmaceuticals, the company plans to file an Investigational New Drug application with the FDA later in the year to launch a phase 1 clinical trial for OR-499.


Orphagen Pharmaceuticals receives rare pediatric disease designation from FDA for OR-499 for the treatment of pediatric adrenocortical carcinoma. Orphagen Pharmaceuticals. January 17, 2023. Accessed January 24, 2023. https://www.orphagen.com/orphagen-pediatric-adrenocortical-carcinoma/

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