NR5A1

SAN DIEGO--(BUSINESS WIRE)-- 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, today 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 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.” About ACC and SF-1 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 10-20%. The estimated annual incidence of ACC in the U.S. 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. Rare Pediatric Disease Designation A rare pediatric disease is defined by the Federal Food, Drug, and Cosmetic Act to include a serious or life-threatening disease, which primarily affects individuals aged from birth to 18 years and affects fewer than 200,000 people in the U.S. To address the challenges that drug companies face when developing treatments for these unique patient populations, Congress reauthorized the Creating Hope Act. Since first enacted by Congress in 2012, the Creating Hope Act has been extended multiple times, most recently on December 27, 2020. Under this Act, companies are eligible to receive a Priority Review Voucher (PRV) following approval of a product with an RPDD 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. If issued, a sponsor may redeem a PRV for priority review of a subsequent marketing application for a different product candidate, or the PRV can be sold or transferred to another sponsor. About Orphagen Pharmaceuticals Orphagen unlocks the power of orphan nuclear receptors. Our scientists excel in discovery and development of small molecule ligands to these largely unexplored drug targets. Starting with a therapeutic area agnostic approach, we are exploring proprietary lead molecules with potential in autoimmune disease and oncology. Orphagen successfully partnered its first program for ROR-gamma antagonists with a mid-size pharmaceutical company ahead of all competitors in the field. Funding from its partnerships and other non-dilutive sources, including federal grants, has allowed Orphagen to advance its proprietary first-in-class drug discovery programs, including OR-449 for adrenocortical cancer. For more info, visit .