Advancing New Small Molecule Treatments for Rare Endocrine Disorders

The First Non-Steroidal Potential Therapy for Congenital Adrenal Hyperplasia

Congenital adrenal hyperplasia (CAH) is a rare endocrine disorder that results in the inability to produce the critical ‘stress’ hormone cortisol. Although CAH testing is a part of the newborn screening program, there are currently only steroidal FDA-approved therapies for CAH. CAH is typically treated with chronic, high-dose steroids often resulting in significant side effects. Spruce’s lead product candidate for CAH, Tildacerfont (SPR001), is intended to correct hormone imbalance, giving patients a therapeutic option that treats their underlying disease and reduces their steroid burden.

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