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).
週五，美國食品藥品監督管理局批准了神經分泌生物科學公司（納斯達克：NBIX）的Crenessity（crinecerfont）膠囊和口服溶液，作爲糖皮質激素替代治療的輔助手段，以控制四歲及以上經典先天性腎上腺增生（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是一種罕見的遺傳性疾病，影響腎上腺並使其比正常情況更大。腎上腺位於腎臟上方，生產調節血糖、血壓和其他身體功能的激素。
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另請閱讀：在Luvadaxistat試驗未達標後，神經分泌生物科學將重心轉向新的精神分裂症治療。
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是一種強效特異性的口服促腎上腺皮質激素釋放因數1型受體拮抗劑，是首個也是唯一一個直接減少過量促腎上腺皮質激素和下遊腎上腺雄激素生成的經典CAH治療藥物，從而能夠減少糖皮質激素的用量。
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預計將在大約一週內上市。爲了集中和簡化處方的履行，該藥物將通過PANTHERx Rare這一專業藥房提供。
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認爲，CAH市場潛力巨大，但也承認要想將其發展成超過10億美元的重大商業機會，必須在患者和臨牀醫生的教育方面做出努力，使得超生理（超過人體正常水平）糖皮質激素用量不再是控制雄激素水平和防止腎上腺危機的唯一方法。
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.
分析師寫道，專注於教育倡議以及與患者倡導團體如CARES基金會的合作，是神經分泌生物科學準備推出其產品的明智之舉。
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.
雖然早期競爭者如Crinetics Pharmaceuticals, Inc（納斯達克：CRNX）顯示出有前景的數據，但神經分泌生物科學作爲首個上市治療先天性腎上腺增生的產品，擁有強勁的競爭優勢。
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.
儘管投資者對crinecerfont的潛在成功意見不一，但William Blair認爲它有機會與Ingrezza一起在長期內成爲一款超級暢銷藥物。
The analyst maintains the Outperform rating on Neurocrine.
分析師維持對神經分泌生物科學的跑贏大盤評級。
Price Action: NBIX stock is up 2.80% at $130.25 premarket at last check Monday.
價格走勢：截至週一最後檢查，NBIX股票在盤前上漲2.80%，報130.25美元。
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Photo via Shutterstock.
圖片來自Shutterstock。
