Bullous Dystrophy, Hereditary Macular Type

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 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. Living with EB entails daily challenges to navigate, including slow-healing wounds at risk of infection and painful dressing changes. 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 "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." 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. About Chiesi Global Rare Diseases Chiesi 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. 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. Filsuvez. Package Insert. Chiesi; 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: 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: 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 Garrick N. Epidermolysis Bullosa. National Institute of Arthritis and Musculoskeletal and Skin Diseases. Published April 11, 2017. Accessed January 26, 2024. 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 For more information, please contact marketing@pantherxrare.com SOURCE PANTHERx Rare

The FDA's approval of rare skin disease drug Filsuvez gives Chiesi Global Rare Diseases its third nod for a new drug from the U.S. regulator this year. Seven months after Krystal Biotech became the first company to gain FDA approval to treat the rare, devastating skin disease epidermolysis bullosa (EB), Chiesi Farmaceutici has followed suit. On Tuesday, the U.S. regulator gave a thumbs up to Chiesi’s Filsuvez (birch triterpenes) to treat both junctional epidermolysis bullosa (JEB) and dystrophic epidermolysis bullosa (DEB) in patients 6 months and older. While Krystal’s topical gene therapy Vyjuvek was endorsed by the FDA in May for DEB, Filsuvez becomes the first FDA-sanctioned treatment for both JEB and DEB. The topical gel treats the wounds associated with EB, a genetic, connective tissue disease that causes the skin to become fragile. Even the slightest friction can tear the skin and cause blisters. EB can be life-threatening as patients are at risk of developing aggressive squamous cell carcinomas and infections. The active ingredient in Filsuvez, which is found in birch trees, accelerates healing and reduces the size of the wound. “The FDA’s decision to approve Filsuvez provides those living with EB a safe and effective treatment option for the most prominent and difficult symptom of EB, open wounds that may not heal,” said Brett Kopelan, the executive director of debra of America, a non-profit that raises money and supports those with EB. This is the third FDA approval this year for Chiesi Global Rare Diseases, which was established in 2020 as one three business units of the family-owned Chiesi Group. The other FDA nods came for enzyme replacement therapies Elfabrio, which treats the neurological disorder Fabry disease, and Lamzede for the genetic condition alpha-mannosidosis, which causes a buildup of sugars in the organs. The FDA green light for Filsuvez was based on the phase 3 EASE trial, which showed significant increase in healing and wound closure compared to patients who received an active control gel, measured at 45 days. Filsuvez was rejected by the FDA in February of 2022, with the regulator asking for more efficacy data. Four months later, Europe endorsed the treatment for DEB and JEB. The drug was owned then by Amryt Pharma of Ireland. In January of this year, Chiesi acquired Amryt Pharma in a $1.25 billion buyout. Along with the purchase came three approved rare disease treatments—Juxtapid, for hypercholerterolemia, Myalept, for lipodystrophy, and Mycapssa, for acromegaly. With its launch of Filsuvez, Chiesi will face formidable competition. Krystal Biotech's launch of Vyvuvek has been so promising that ISI Evercore bumped up its projected 2024 sales of the gene therapy to $273 million. 

Certain children with the rare skin disease epidermolysis bullosa now have a treatment option thanks to an FDA approval for Chiesi Global Rare Diseases’ topical gel Filsuvez. Chiesi said Tuesday the FDA greenlit the gel for treating partial thickness wounds in patients as young as 6 months old who have forms of EB known as junctional (JEB) and dystrophic (DEB). Filsuvez is the first treatment approved for wounds associated with JEB, Chiesi said. Krystal Biotech’s Vyjuvek was approved for the DEB form in May, making it the first gene therapy to be applied topically. You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.