Spruce Biosciences announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for SPR001 for the treatment of congenital adrenal hyperplasia (CAH).
Earlier in 2017, Spruce initiated a Phase 2 clinical trial to assess the safety and efficacy of SPR001 in adults with classic CAH. This study is currently enrolling at centers across the U.S., with topline data expected in 2018. Patients interested in seeing if they may be eligible to participate can visit the clinical trial study site here.
"Receiving orphan drug designation is an important regulatory milestone, and we are pleased that SPR001 for CAH has been granted this status. 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.
CAH is a rare endocrine disorder that is caused by genetic mutations resulting in the inability to produce the critical 'stress' hormone cortisol. Although CAH is part of the newborn screening program, there are currently no FDA-approved therapies for CAH. CAH is typically treated with chronic, high-dose steroids which commonly leads to significant side effects and long-term health consequences.
The FDA grants orphan drug designation to promote the development of promising products for rare conditions that affect fewer than 200,000 people in the U.S. and for which significant unmet need remains. This designation provides sponsors with development and commercial incentives including market exclusivity, tax credits for clinical research costs and the waiver of certain administrative fees.