Orphagen Pharmaceuticals has received a rare pediatric disease designation (RPDD) for OR-449 from the US Food and Drug Administration (FDA) to treat paediatric adrenocortical carcinoma (ACC).

OR-449 is a first-in-class, orally bioavailable, potent and selective small molecule antagonist of the orphan nuclear receptor steroidogenic factor-1 (SF-1, NR5A1).

SF-1 is an important transcription factor for adrenal gland growth and development. It is highly expressed in adult patients with ACC, a rare and aggressive adrenal gland cancer.

The company is developing OR-449 to treat ACC in adult and paediatric patients.

The SF-1 antagonist is also being developed to treat other cancers, which are known to express increased SF-1 levels.

Orphagen Pharmaceuticals CEO Scott Thacher said: “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 the initiation of a Phase I clinical trial.

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“We speak to clinicians regularly who remind us of the urgent need for improved therapy for patients with ACC, and this drives our commitment to the clinical development of OR-449, our first internal programme to reach this stage.”

According to the company, approximately 600 patients per year are reported with ACC in the US.

SF-1 has been recognised as a potential therapeutic target for ACC in adult and paediatric patients and is sad to be widely used as a marker for the indication.

In paediatric ACC, the transcription factor is commonly amplified at the gene level.