Orphagen Pharmaceuticals Receives Rare Pediatric Disease Designation from FDA for OR-449 for the Treatment of Pediatric Adrenocortical Carcinoma

Orphagen Pharmaceuticals, Inc., a biotech company pioneering the screening, discovery, and development of small molecule ligands that modulate orphan or unexplored members of the nuclear receptor family, announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for OR-449 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 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 are gratified that OR-449 has received an RPDD from the FDA. We currently plan to file an Investigational New Drug (IND) application with the FDA later this year to support initiation of a Phase 1 clinical trial,” said Orphagen CEO, Scott Thacher, Ph.D. “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.”