Spruce Biosciences passes US FDA orphan drug designation for SPR001 to treat rare endocrine disease CAH.

M2 PHARMA-December 5, 2017-Spruce Biosciences passes US FDA orphan
drug designation for SPR001 to treat rare endocrine disease CAH

(C)2017 M2 COMMUNICATIONS

Biotechnology company Spruce Biosciences reported on Monday the
receipt of the US Food and Drug Administration (FDA) orphan drug
designation for SPR001 for the treatment of congenital adrenal
hyperplasia (CAH).

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 programme, there are currently no US FDA-approved therapies
for CAH. CAH is typically treated with chronic, high-dose steroids.

Early in 2017, the company launched a Phase 2 clinical trial to
assess the safety and efficacy of SPR001 in adults with classic CAH.

This Phase 2 study is currently enrolling at centres across the US,
with topline data expected in 2018. Eligible patients can visit the
clinical trial study site.

The US FDA reportedly grants orphan drug designation to promote the
development of promising products for rare conditions that affect fewer
than 200,000 people in the US 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.

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