FDA Approves BridgeBio Pharma's Drug For Rare Heart Disease, Poised To Challenge Pfizer In Lucrative Yet Competitive Market
FDA Approves BridgeBio Pharma's Drug For Rare Heart Disease, Poised To Challenge Pfizer In Lucrative Yet Competitive Market
Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR.On Friday, the FDA approved BridgeBio Pharma, Inc.'s (NASDAQ:BBIO) Attruby (acoramidis), an orally-administered near-complete (≥90%) stabilizer of Transthyretin (TTR) for adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization.
上週五，FDA批准了BridgeBio Pharma, Inc.(納斯達克:BBIO)的Attruby (acoramidis)，一種口服近全（≥90%）轉甲烯基甲基雙胍烷基甲酸酯穩定劑，用於減少ATTR-Cm成人的心血管死亡和心血管相關住院。
The FDA approval is based on ATTRibute-CM Phase 3 study results, which showed that Attruby significantly reduced death and cardiovascular-related hospitalization and improved quality of life.
FDA的批准基於ATTRibute-Cm第3期研究結果，顯示Attruby顯著減少死亡和心血管相關住院，並改善生活質量。
Also Read: BridgeBio Pharma's Acoramidis Shows Competitive Edge Against Pfizer's Tafamidisa In Lucrative But Competitive ATTR-CM Market
另請參閱：BridgeBio Pharma的Acoramidis在豐厚但具競爭力的ATTR-Cm市場中顯示出與輝瑞的Tafamidisa具有競爭優勢。
Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR.
Attruby是唯一獲批准且具有標籤指定的近全TTR穩定化的產品。
Attruby was designed to mimic a naturally occurring "rescue mutation" of the TTR gene (T119M) that targets the root cause of ATTR-CM, the destabilization of the native TTR tetramer.
Attruby的設計目的是模擬TTR基因（T119M）的自然發生的「拯救突變」，針對ATTR-Cm的病因，即TTR四聚體的不穩定。
Through near-complete TTR stabilization, Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and to demonstrate benefit on cardiovascular outcomes.
通過近全TTR穩定化，Attruby已被證明保留TTR作爲甲狀腺素和維生素A的運輸蛋白的天然功能，並對心血管結果產生益處。
The ATTRibute-CM Phase 3 study enrolled 632 participants with symptomatic ATTR-CM associated with either wild-type or variant TTR.
ATTRibute-Cm第3期研究招募了632名與野生型或變異型TTR相關的症狀性ATTR-Cm參與者。
BridgeBio submitted a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025.
BridgeBio已向歐洲藥品管理局遞交了營銷授權申請，預計將在2025年作出決定。
BridgeBio has granted Bayer AG (OTC:BAYRY) exclusive rights to commercialize acoramidis for ATTR-CM in Europe.
BridgeBio已授予拜耳（adr）在歐洲商業化acoramidis用於ATTR-Cm的獨家權利。
The company said it will provide Attruby for free to patients who participated in the trial.
該公司表示將爲參與試驗的患者免費提供 Attruby。
William Blair writes, "We continue to view a reduction in all-cause mortality on label as a necessary distinction to gain major market share in ATTR-CM alongside Pfizer Inc's (NYSE:PFE) tafamidis."
威廉·布萊爾（William Blair）表示：「我們仍然認爲，在ATTR-Cm領域，以所有原因死亡率減少作爲必要的區分標準，是輝瑞公司(NYSE:PFE)塔法米迪絲獲得主要市場份額的關鍵。」
Since the Attruby label doesn't include data on overall mortality, the William Blair analyst notes Pfizer might shift its marketing focus to emphasize claims about mortality benefits.
由於 Attruby 產品標籤不包含關於整體死亡率的數據，威廉·布萊爾分析師指出，輝瑞可能會轉移其營銷重點，強調有關死亡率益處的聲明。
Price Action: BBIO stock is up 24.3% at $29.10 during the premarket session at last check Monday.
股價走勢：BBIO股票在週一最後一次查詢市前交易時上漲了24.3%，報29.10美元。
Image via Shutterstock
圖片來源：shutterstock
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