San Diego-based Neurocrine Biosciences Inc. announced Monday its drug for treating a rare disease of the adrenal glands was granted “orphan” status by the Food and Drug Administration to encourage its development and marketing.
Neurocrine’s NBI-77860 is being tested for treatment of congenital adrenal hyperplasia, or CAH, a disease that affects approximately 20,000 to 30,000 people in the United States.
“We are very pleased that the FDA has granted NBI-77860 orphan status to treat congenital adrenal hyperplasia, a devastating disease that is a significant challenge for both clinicians and patients,” said Malcolm Lloyd-Smith, chief regulatory officer of Neurocrine.
“This status represents a significant regulatory milestone for the CAH program and underscores the importance of bringing a safe and effective CAH therapy to market.”
Orphan drug designation is granted by the FDA for medicines for disorders that affect fewer than 200,000 people in the United States. The designation provides companies with development and commercial incentives.
CAH is a genetic disorder that results in an enzyme deficiency altering the production of adrenal steroids. Because of this deficiency, the adrenal glands have little to no cortisol biosynthesis resulting in a potentially life-threatening condition. If left untreated, classic CAH can result in salt wasting, dehydration and eventually death.
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