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
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.
"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.
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.
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.
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
SOURCE PANTHERx Rare Pharmacy
匹兹堡，2024年11月26日 / PRNewswire / - PANTHERx Rare是美国罕见疾病产品患者获取和支持服务领域的领先者，很高兴宣布被BridgeBio选中分销Attruby（acoramidis），这是一种高亲和性甲状腺素转甲胺蛋白（TTR）稳定剂，每天口服两次，已获批用于治疗甲状腺素转甲胺蛋白心肌病（ATTR-CM）。2,5
Attruby是第一款和唯一一款具有特定标签，指明几乎完全稳定TTR的批准产品。Attruby的设计目的是模仿TTR基因（T119M）的天然“拯救突变”，针对ATTR-CM的根本原因，即原生TTR四聚体的不稳定。通过几乎完全的TTR稳定性，已经表明Attruby保留了TTR作为甲状腺素和维生素A的运输蛋白的天然功能，并对心血管结果产生益处。
“Panther致力于为罕见且具有毁灭性疾病的患者提供量身定制的解决方案，我们期待与BridgeBio的合作，并为患有甲状腺素转甲胺蛋白心肌病的患者提供服务，”PANTHERx Rare Pharmacy的执行主席Rob Snyder表示。
关于PANTHERx Rare
PANTHERx Rare是一家致力于罕见和孤儿病专业药房，拥有两项认证，分发多种孤儿产品，并为最需要的人提供获得和支持服务。 PANTHERx通过提供医学突破、临床卓越和获得解决方案，改变生活，助力患有罕见且具有毁灭性疾病的人。尽管每种罕见病影响的人数很少，但所有罕见疾病共影响了大约2500万至3000万美国人。目前已经确定了超过7,000种罕见疾病，超过90%的罕见疾病仍没有经过FDA批准的治疗方法。3 联邦政策的变化和科学的进步导致FDA孤儿药物批准激增，为罕见疾病社区提供了巨大的希望。
PANTHERx是一家双重认证的专业药店，在罕见疾病领域获得了多项荣誉，包括医疗保健委员会（ACHC）孤儿药和利用评审委员会（URAC）稀有疾病卓越中心。 作为一家专注于患者满意度的药店，PANTHERx已经六次荣获著名的MMIt患者选择奖，包括2023年的荣誉。PANTHERx总部设在宾夕法尼亚州匹兹堡，并在所有50个州和美国领土获得许可。
1.转甲状腺前白蛋白淀粉样心肌病（ATTR-CM）。于2024年10月9日查阅。
2. Acoramidis（AG10）TTR稳定剂用于ATTR。Eidos，TX。bridgebiowp。于2024年10月9日查阅。
3. 什么是ATTR？| TTR生命周期| Eidos TX。BridgeBio。于2024年10月9日查阅。
4.在疾病进展之前诊断遗传性ATTR淀粉样病。于2024年10月9日查阅。
5. Acoramidis。包装说明书。BridgeBio；2024
来源：PANTHERx罕见药物药店
