BridgeBio Selects PANTHERx Rare Specialty Pharmacy for Distribution of Attruby (Acoramidis)
BridgeBio Selects PANTHERx Rare Specialty Pharmacy for Distribution of Attruby (Acoramidis)
PITTSBURGH, Nov. 26, 2024 /PRNewswire/ -- PANTHERx Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce that it was selected by BridgeBio for the distribution of Attruby (acoramidis), a high-affinity transthyretin (TTR) stabilizer for twice daily oral administration, approved for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).2,5
匹茲堡，2024年11月26日 / PRNewswire / - PANTHERx Rare是美國罕見疾病產品患者獲取和支持服務領域的領先者，很高興宣佈被BridgeBio選中分銷Attruby（acoramidis），這是一種高親和性甲狀腺素轉甲胺蛋白（TTR）穩定劑，每天口服兩次，已獲批用於治療甲狀腺素轉甲胺蛋白心肌病（ATTR-CM）。2,5
Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. Attruby was designed to mimic a naturally occurring "rescue mutation" of the TTR gene (T119M) that targets the root cause of ATTR-CM, destabilization of the native TTR tetramer. 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.
Attruby是第一款和唯一一款具有特定標籤，指明幾乎完全穩定TTR的批准產品。Attruby的設計目的是模仿TTR基因（T119M）的天然「拯救突變」，針對ATTR-CM的根本原因，即原生TTR四聚體的不穩定。通過幾乎完全的TTR穩定性，已經表明Attruby保留了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。
"Panther is committed to providing tailored solutions for people living with rare and devastating conditions and we look forward to the partnership with BridgeBio and serving the needs of patients suffering from transthyretin amyloid cardiomyopathy," said Rob Snyder, Executive Chair, PANTHERx Rare Pharmacy.
「Panther致力於爲罕見且具有毀滅性疾病的患者提供量身定製的解決方案，我們期待與BridgeBio的合作，併爲患有甲狀腺素轉甲胺蛋白心肌病的患者提供服務，」PANTHERx Rare Pharmacy的執行主席Rob Snyder表示。
About PANTHERx Rare
PANTHERx Rare is a dual-accredited specialty pharmacy focused on rare and orphan disease, distributing several orphan products and providing access and support services to those needing them most. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. While each rare condition affects few people, together all rare diseases impact an estimated 25 to 30 million Americans.3 Currently over 7,000 rare diseases have been identified and more than 90% of rare diseases are still without an FDA-approved treatment. 3 Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.
關於PANTHERx Rare
PANTHERx Rare是一家致力於罕見和孤兒病專業藥房，擁有兩項認證，分發多種孤兒產品，併爲最需要的人提供獲得和支持服務。 PANTHERx通過提供醫學突破、臨床卓越和獲得解決方案，改變生活，助力患有罕見且具有毀滅性疾病的人。儘管每種罕見病影響的人數很少，但所有罕見疾病共影響了大約2500萬至3000萬美國人。目前已經確定了超過7,000種罕見疾病，超過90%的罕見疾病仍沒有經過FDA批准的治療方法。3 聯邦政策的變化和科學的進步導致FDA孤兒藥物批准激增，爲罕見疾病社區提供了巨大的希望。
PANTHERx is a dual-accredited specialty pharmacy, holding distinctions in rare disease including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence.4-5 As a pharmacy focused on patient satisfaction, PANTHERx is now a six-time winner of the prestigious MMIT Patient Choice Award, including the 2023 honor.6 PANTHERx is headquartered in Pittsburgh, Pennsylvania and is licensed in all 50 states and US territories.
PANTHERx是一家雙重認證的專業藥店，在罕見疾病領域獲得了多項榮譽，包括醫療保健委員會（ACHC）孤兒藥和利用評審委員會（URAC）稀有疾病卓越中心。 作爲一家專注於患者滿意度的藥店，PANTHERx已經六次榮獲著名的MMIt患者選擇獎，包括2023年的榮譽。PANTHERx總部設在賓夕法尼亞州匹茲堡，並在所有50個州和美國領土獲得許可。
1.Transthyretin Amyloid Cardiomyopathy (ATTR-CM). . Accessed on October 9, 2024.
2. Acoramidis (AG10) TTR Stabilizer For ATTR. Eidos, TX. bridgebiowp. Accessed on October 9, 2024.
3. What is ATTR? | TTR Life-Cycle | Eidos TX. BridgeBio. Accessed on October 9, 2024.
4. Diagnose hATTR Amyloidosis Before Disease Progression. Accessed on October 9, 2024. .
5. Acoramidis. Package Insert. BridgeBio; 2024
1.轉甲狀腺前白蛋白澱粉樣心肌病（ATTR-CM）。於2024年10月9日查閱。
2. Acoramidis（AG10）TTR穩定劑用於ATTR。Eidos，TX。bridgebiowp。於2024年10月9日查閱。
3. 什麼是ATTR？| TTR生命週期| Eidos TX。BridgeBio。於2024年10月9日查閱。
4.在疾病進展之前診斷遺傳性ATTR澱粉樣病。於2024年10月9日查閱。
5. Acoramidis。包裝說明書。BridgeBio；2024
SOURCE PANTHERx Rare Pharmacy
來源：PANTHERx罕見藥物藥店
譯文內容由第三人軟體翻譯。
