Neurocrine's Drug Gains FDA Nod As First Targeted Treatment For Certain Type Of Inherited Disorder, Analyst Highlights Blockbuster Potential
Neurocrine's Drug Gains FDA Nod As First Targeted Treatment For Certain Type Of Inherited Disorder, Analyst Highlights Blockbuster Potential
Crenessity, a potent and selective oral corticotropin-releasing factor type 1 receptor antagonist, is the first and only classic CAH treatment that directly reduces excess adrenocorticotropic hormone and downstream adrenal androgen production, allowing for glucocorticoid dose reduction.On Friday, the FDA approved Neurocrine Biosciences, Inc.(NASDAQ:NBIX) Crenessity (crinecerfont) capsules and oral solution as an adjunctive treatment to glucocorticoid replacement to control androgens in adult and pediatric patients four years of age and older with classic congenital adrenal hyperplasia (CAH).
周五，美国食品药品监督管理局批准了神经分泌生物科学公司（纳斯达克：NBIX）的Crenessity（crinecerfont）胶囊和口服溶液，作为糖皮质激素替代治疗的辅助手段，以控制四岁及以上经典先天性肾上腺增生（CAH）患者的雄激素。
CAH is a rare, inherited disorder that affects the adrenal glands and causes them to be larger than normal. The adrenal glands are located above the kidneys and produce hormones that regulate blood sugar, blood pressure, and other bodily functions.
CAH是一种罕见的遗传性疾病，影响肾上腺并使其比正常情况更大。肾上腺位于肾脏上方，生产调节血糖、血压和其他身体功能的激素。
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Crenessity, a potent and selective oral corticotropin-releasing factor type 1 receptor antagonist, is the first and only classic CAH treatment that directly reduces excess adrenocorticotropic hormone and downstream adrenal androgen production, allowing for glucocorticoid dose reduction.
Crenessity是一种强效特异性的口服促肾上腺皮质激素释放因子1型受体拮抗剂，是首个也是唯一一个直接减少过量促肾上腺皮质激素和下游-脑机肾上腺雄激素生成的经典CAH治疗药物，从而能够减少糖皮质激素的用量。
Crenessity is expected to be commercially available in approximately one week. To centralize and simplify prescription fulfillment, the medication will be provided through PANTHERx Rare, a specialty pharmacy.
Crenessity预计将在大约一周内上市。为了集中和简化处方的履行，该药物将通过PANTHERx Rare这一专业药房提供。
William Blair sees significant potential in the CAH market but acknowledges that for this to be a blockbuster $1 billion-plus commercial opportunity, there will need to be both patient and clinician education that supraphysiological (amounts greater than normally found in the body) glucocorticoid dosing would no longer be the only way to control androgen levels and prevent adrenal crises.
William Blair认为，CAH市场潜力巨大，但也承认要想将其发展成超过10亿美元的重大商业机会，必须在患者和临床医生的教育方面做出努力，使得超生理（超过人体正常水平）糖皮质激素用量不再是控制雄激素水平和防止肾上腺危机的唯一方法。
The analyst writes that focusing on educational initiatives and collaborating with patient advocacy groups, such as the CARES Foundation, is a smart move as Neurocrine prepares for its product launch.
分析师写道，专注于教育倡议以及与患者倡导团体如CARES基金会的合作，是神经分泌生物科学准备推出其产品的明智之举。
While early-stage competitors like Crinetics Pharmaceuticals, Inc (NASDAQ:CRNX) show promising data, Neurocrine's position as the first to market in treating congenital adrenal hyperplasia gives it a strong advantage.
虽然早期竞争者如Crinetics Pharmaceuticals, Inc（纳斯达克：CRNX）显示出有前景的数据，但神经分泌生物科学作为首个上市治疗先天性肾上腺增生的产品，拥有强劲的竞争优势。
Although investors have mixed opinions about the potential success of crinecerfont, William Blair sees opportunities for it to become a blockbuster drug alongside Ingrezza in the long run.
尽管投资者对crinecerfont的潜在成功意见不一，但William Blair认为它有机会与Ingrezza一起在长期内成为一款超级畅销药物。
The analyst maintains the Outperform rating on Neurocrine.
分析师维持对神经分泌生物科学的跑赢大盘评级。
Price Action: NBIX stock is up 2.80% at $130.25 premarket at last check Monday.
价格走势：截至周一最后检查，NBIX股票在盘前上涨2.80%，报130.25美元。
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