The Food and Drug Administration (FDA) has granted orphan drug designation to SPR001 (Spruce Biosciences), a novel treatment intended for patients with congenital adrenal hyperplasia (CAH).

There is currently no FDA-approved treatment for CAH; typical treatment includes chronic, high-dose steroids which could potentially lead to long-term health consequences. CAH is a rare endocrine disorder caused by genetic mutations which result in an inability to produce cortisol.

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Spruce Biosciences is currently enrolling adults with classic CAH in a Phase 2 study to assess the safety and efficacy of SPR001. Topline data are expected in 2018.

“We are developing SPR001 with the objective of providing individuals with CAH an effective treatment, and our team is committed to advancing the clinical development program to address this unmet need,” said Dr. Alexis Howerton, CEO, Spruce Biosciences.

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