PITTSBURGH, Feb. 5, 2024 /PRNewswire/ -- PANTHERx® Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce it was selected by Chiesi Global Rare Diseases, a business unit of the Chiesi Group, for the distribution of Filsuvez® topical gel, the first approved treatment for wounds associated with both dystrophic and junctional epidermolysis bullosa (EB) in adult and pediatric patients 6 months of age and older.1.

匹兹堡，2024年2月5日/PRNewswire/--PANTHERx®Rare是美国罕见病产品患者获取和支持服务的领导者，很高兴地宣布它被Chiesi集团的业务部门Chiesi Global Rare Diseases选中，用于分销Filsuvez®外用凝胶，首次批准治疗6个月及以上的成人和儿科患者营养不良和交界性大疱性表皮松解症（EB）相关的伤口。

EB is a group of inherited disorders characterized by fragile skin and mucous membranes that result in formation of blisters and wounds in response to minor trauma and can lead to fibrosis and scarring, joint contracture, and increased risk for developing cutaneous squamous cell carcinoma (SCC).2-4 Dystrophic epidermolysis bullosa (DEB) is a major type of EB resulting from impaired type VII collagen expression which leads to separation of skin layers between the basement membrane and the upper dermis.3,5 Junctional epidermolysis bullosa (JEB) is a less common subtype of EB that occurs from various absent or deficient adhesion proteins leading to separation of the epidermis and basement membrane.3,5 Both are associated with intolerable pain with limited mobility.

EB是一组遗传性疾病，其特征是皮肤和粘膜脆弱，在轻微创伤下会导致水疱和伤口的形成，并可能导致纤维化和瘢痕形成，关节挛缩，2-4营养不良性大疱性表皮松解症（DEB）是EB的主要类型，是由于VII型胶原表达受损导致的，导致基底膜和真皮上层之间的皮肤层分离。3,5交界性大疱性表皮松解症（JEB）是EB的一种不太常见的亚型，发生于各种缺失或缺乏粘附蛋白导致表皮和基底膜分离[3,5]。两者都与活动受限的不可忍受的疼痛有关。

Living with EB entails daily challenges to navigate, including slow-healing wounds at risk of infection and painful dressing changes..

与EB一起生活需要每天应对挑战，包括有感染风险的伤口愈合缓慢和痛苦的换药。。

Filsuvez is applied topically to the wound surface at home, during wound dressing changes and works to accelerate wound healing with a goal of achieving complete wound closure within 45 days.6

在伤口敷料更换期间，将Filsuvez局部应用于家中的伤口表面，并加速伤口愈合，目标是在45天内实现完全伤口闭合

'We strive to be the catalyst that seamlessly delivers essential medications to individuals confronting rare conditions,' said Rob Snyder, CEO of PANTHERx® Rare Pharmacy. 'We look forward to working with the Chiesi Global Rare Diseases to make a difference in the lives of those living with wounds associated with both dystrophic and junctional epidermolysis bullosa.'.

PANTHERx®rare Pharmacy首席执行官罗伯·斯奈德（RobSnyder）说：“我们努力成为催化剂，为面临罕见疾病的个体无缝提供基本药物。”我们期待着与Chiesi全球罕见疾病合作，改变那些患有营养不良和交界性大疱性表皮松解症的伤口的人的生活。”。

About PANTHERx RarePANTHERx 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.

关于PANTHERx RarePANTHERx Rare是一家双重认证的专业药房，专注于罕见病和孤儿病，分销几种孤儿产品，并为最需要的人提供获取和支持服务。PANTHERx通过为患有罕见和毁灭性疾病的人提供医学突破、临床卓越和获取解决方案来改变生活。

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..

虽然每种罕见病影响的人很少，但所有罕见病加在一起估计会影响2500万至3000万美国人。3目前，已发现7000多种罕见病，超过90%的罕见病仍未得到FDA批准的治疗。3联邦政策的变化和科学的进步导致FDA孤儿药批准激增，为罕见病社区带来巨大希望。。

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是一家获得双重认证的专业药房，在罕见病方面拥有多项殊荣，包括孤儿药卫生保健认证委员会（ACHC）和利用审查认证委员会（URAC）罕见病卓越中心。4-5作为一家专注于患者满意度的药房，PANTHERx目前已六次荣获著名的MMIT患者选择奖，包括2023年荣誉。6黑豹总部位于宾夕法尼亚州匹兹堡，在美国所有50个州和地区获得许可。。

About Chiesi Global Rare DiseasesChiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet.

关于Chiesi全球罕见病Chiesi Global Rare Diseases是Chiesi Group的一个业务部门，旨在为受罕见病影响的人群提供创新疗法和解决方案。作为一家家族企业，Chiesi Group致力于创造一个普遍治疗所有疾病的世界，并成为造福社会和地球的力量。

The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system..

全球罕见病股的目标是确保平等获取，使尽可能多的人能够体验最充实的生活。该部门与全球罕见病社区合作，为医疗保健系统中服务不足的人发出声音。。

Filsuvez. Package Insert. Chiesi; 2023.

菲尔苏维斯。包装插页。基耶西；2023

Kern JS, Sprecher E, Fernandez MF, et al. Efficacy and safety of Oleogel-S10 (birch triterpenes) for epidermolysis bullosa: results from the phase III randomized double-blind phase of the EASE study. British Journal of Dermatology. 2022;188(1):12-21. doi:https://doi.org/10.1093/bjd/ljac001

Kern JS，Sprecher E，Fernandez MF等。油凝胶-S10（桦木三萜）治疗大疱性表皮松解症的疗效和安全性：EASE研究的III期随机双盲阶段的结果。英国皮肤病学杂志。2022年；188（1）：12-21。内政部：https://doi.org/10.1093/bjd/ljac001

Štublar A, Dragoš V, Dolenc-Voljč M. Inherited epidermolysis bullosa: epidemiology and patient care in Slovenia with a review of the updated classification. Acta Dermatovenerologica Alpina Pannonica et Adriatica. 2021;30(2). doi:https://doi.org/10.15570/actaapa.2021.15

Štublar A，DragošV，Dolenc VoljčM.遗传性大疱性表皮松解症：斯洛文尼亚的流行病学和患者护理，并回顾了最新的分类。皮肤性病学报Alpina Pannonica et Adriatica。2021年；30（2）。内政部：https://doi.org/10.15570/actaapa.2021.15

Boeira VL, Souza ES, Rocha Bde O, et al. Inherited epidermolysis bullosa: clinical and therapeutic aspects. An Bras Dermatol. 2013;88(2):185-198. doi:10.1590/S0365-05962013000200001

Boeira VL，Souza ES，Rocha Bde O等。遗传性大疱性表皮松解症：临床和治疗方面。胸罩皮肤病。2013年；88（2）：185-198。内政部：10.1590/S0365-059620130000001

Garrick N. Epidermolysis Bullosa. National Institute of Arthritis and Musculoskeletal and Skin Diseases. Published April 11, 2017. Accessed January 26, 2024. https://www.niams.nih.gov/health-topics/epidermolysis-bullosa

加里克N.大疱性表皮松解症。国家关节炎、肌肉骨骼和皮肤病研究所。2017年4月11日发布。2024年1月26日访问https://www.niams.nih.gov/health-topics/epidermolysis-bullosa

Hou PC, Del Agua N, Lwin SM, Hsu CK, McGrath JA. Innovations in the Treatment of Dystrophic Epidermolysis Bullosa (DEB): Current Landscape and Prospects. Ther Clin Risk Manag. 2023;19:455-473. Published 2023 Jun 14. doi:10.2147/TCRM.S386923

侯PC，德尔阿古N，Lwin SM，Hsu CK，McGrath JA。营养不良性大疱性表皮松解症（DEB）治疗的创新：现状和前景。临床风险管理。2023年；19： 455-473。2023年6月14日出版。doi:10.2147/TCRM.S386923

For more information, please contact marketing@pantherxrare.com

有关更多信息，请联系marketing@pantherxrare.com

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