Rare Disease Report

Spruce Receives Orphan Drug Designation for Treatment of Endocrine Disease

DECEMBER 04, 2017
Mathew Shanley
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to SPR001, a novel therapy being developed by Spruce Biosciences for the potential treatment of congenital adrenal hyperplasia (CAH).

The designation will provide Spruce Biosciences and its sponsors with development and commercial incentives including market exclusivity, tax credits for clinical research costs and the waiver of certain administrative fees.

CAH, a rare endocrine disease, is the result of a genetic mutation in the enzyme responsible for the synthesis of cortisols. It is characterized by overgrowth of the adrenal glands, adrenal insufficiency, mineralocorticoid deficiency, and androgen excess. While the condition is tested for via newborn screening, there aren’t currently any approved therapies for it, and it is typically treated with chronic, high-dose steroids which commonly lead to significant side effects and long-term health consequences.

The most common symptom in patients with CAH is severe dehydration, however, other symptoms can include fluctuation in weight, vomiting, abnormal growth patterns, and acne.

Earlier in 2017, Spruce initiated a Phase 2 clinical trial designed to evaluate the safety and efficacy of SPR001 in adult patients with classic CAH. The multicenter dose escalation study expects to publish topline data in 2018.

"Receiving orphan drug designation is an important regulatory milestone, and we are pleased that SPR001 for CAH has been granted this status,” said Dr. Alexis Howerton, CEO, Spruce Biosciences in a press release. “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.”

An estimated 35-40,000 people are affected by CAH in the U.S. The disease is defined as either “classical” or “non-classical,” and the two types are differentiated by time of diagnosis. Classical CAH is present at birth and can be life-threatening if not properly diagnosed. Non-classical CAH is less severe and typically begins to present during puberty.

"Treatments to improve the lives of CAH patients are of critical interest to CARES Foundation. We applaud the work of Spruce Biosciences in this space and look forward to continuing to collaborate with Spruce to provide patients with more options," added Dina Matos, Executive Director, CARES Foundation. CARES is a non-profit organization dedicated to improving the lives of patients with CAH through community support, advocacy, education, and research.

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