Clinical data | |
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Trade names | Filsuvez |
Other names | Oleogel-S10 |
AHFS/Drugs.com | Monograph |
MedlinePlus | a624016 |
License data | |
Routes of administration | Topical |
ATC code | |
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Birch triterpenes, sold under the brand name Filsuvez, is an extract of birch bark used as a topical medication for the treatment of epidermolysis bullosa. [1] [3] The active ingredients are triterpenes extracted from the outer bark of silver birch (Betula pendula) and downy birch (Betula pubescens). [4]
The most common side effects include wound complications such as skin reactions at the application site, infections, pruritus (itching), and hypersensitivity. [3]
Birch triterpenes was approved for medical use in the European Union in June 2022, [3] and in the United States in December 2023. [5] [6]
Epidermolysis bullosa is an inherited disease of the skin that makes the skin very fragile and causes severe blistering and scarring. [3] Birch triterpenes is used in two types of epidermolysis bullosa, dystrophic epidermolysis bullosa and junctional epidermolysis bullosa, to treat partial-thickness skin wounds. [3] These are wounds where the upper layers of the skin have been damaged. [3]
Birch triterpenes is indicated for the treatment of partial thickness wounds associated with dystrophic and junctional epidermolysis bullosa in people aged six months of age and older. [1] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [7]
The most common side effects include wound complications. [3] Other common side effects include skin reactions at the application site, wound infections, pruritus (itching), and hypersensitivity (allergic) reactions. [3]
The active substance of birch triterpenes is birch bark extract (as dry extract, refined) from Betula pendula Roth/ Betula pubescens Ehrh. (equivalent to 0.5‑1.0 g birch bark), including 84‑95 mg triterpenes calculated as the sum of betulin, betulinic acid, erythrodiol, lupeol, and oleanolic acid. [3] The mechanism of action of birch triterpenes in the treatment of wounds associated with epidermolysis bullosa is unknown. [1] It is thought to work by modulating inflammatory mediators and stimulating keratinocyte differentiation and migration, thereby promoting wound healing and closure. [3]
The pharmacodynamics of birch triterpenes are unknown. [1]
The US FDA approved birch triterpenes based on evidence from a clinical trial (NCT03068780 [1] [8] ) of 223 participants with dystrophic and junctional epidermolysis bullosa. [6] The efficacy and safety of birch triterpenes for the treatment of partial-thickness wounds associated with inherited epidermolysis bullosa was evaluated in a randomized, double-blind, placebo-controlled trial in participants aged six months of age and older with dystrophic epidermolysis bullosa and junctional epidermolysis bullosa. [6] The primary endpoint was the proportion of subjects with first complete closure of the target wound by day 45 of the 90-day double-blind phase of the study, based on clinical assessment by the investigator. [6] The trial was conducted at 49 sites in 26 countries including Argentina, Australia, Austria, Belgium, Brazil, Chile, Colombia, Croatia, the Czech Republic, Denmark, France, Germany, Greece, Hong Kong, Hungary, Ireland, Israel, Italy, Romania, Russia, Serbia, Singapore, Spain, Switzerland, the United Kingdom, and the United States. [6] There were 14 participants enrolled in the United States and 209 participants were enrolled outside the United States. [6]
In April 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Filsuvez, intended for the treatment of epidermolysis bullosa. [9] The applicant for this medicinal product is Amryt Pharmaceuticals DAC. [9] Birch triterpenes was approved for medical use in the European Union in June 2022, [3] [10] and in the United States in December 2023. [5] [11] [12]
A birch is a thin-leaved deciduous hardwood tree of the genus Betula, in the family Betulaceae, which also includes alders, hazels, and hornbeams. It is closely related to the beech-oak family Fagaceae. The genus Betula contains 30 to 60 known taxa of which 11 are on the IUCN 2011 Red List of Threatened Species. They are typically short-lived pioneer species and are widespread in the Northern Hemisphere, particularly in northern areas of temperate climates and in boreal climates. Birch wood is used for a wide range of purposes.
Betula pendula, commonly known as silver birch, warty birch, European white birch, or East Asian white birch, is a species of tree in the family Betulaceae, native to Europe and parts of Asia, though in southern Europe, it is only found at higher altitudes. Its range extends into Siberia, China, and southwest Asia in the mountains of northern Turkey, the Caucasus, and northern Iran. It has been introduced into North America, where it is known as the European white birch or weeping birch and is considered invasive in some states in the United States and parts of Canada. The tree can also be found in more temperate regions of Australia.
Epidermolysis bullosa (EB) is a group of rare medical conditions that result in easy blistering of the skin and mucous membranes. Blisters occur with minor trauma or friction and are painful. Its severity can range from mild to fatal. Inherited EB is a rare disease with a prevalence in the United States of 8.2 per million live births. Those with mild cases may not develop symptoms until they start to crawl or walk. Complications may include esophageal narrowing, squamous cell skin cancer, and the need for amputations.
DEBRA is the name of an international medical research charity dedicated to securing effective drug treatments and ultimately cures for every type of epidermolysis bullosa, with national groups in over 40 countries including in the United Kingdom and the United States.
Birch bark or birchbark is the bark of several Eurasian and North American birch trees of the genus Betula.
Ustekinumab, sold under the brand name Stelara among others, is a monoclonal antibody medication developed by Janssen Pharmaceuticals, for the treatment of Crohn's disease, ulcerative colitis, plaque psoriasis and psoriatic arthritis, targeting both IL-12 and IL-23.
Epidermolysis bullosa dystrophica or dystrophic EB (DEB) is an inherited disease affecting the skin and other organs.
Collagen alpha-1(VII) chain is a protein that in humans is encoded by the COL7A1 gene. It is composed of a triple helical, collagenous domain flanked by two non-collagenous domains, and functions as an anchoring fibril between the dermal-epidermal junction in the basement membrane. Mutations in COL7A1 cause all types of dystrophic epidermolysis bullosa, and the exact mutations vary based on the specific type or subtype. It has been shown that interactions between the NC-1 domain of collagen VII and several other proteins, including laminin-5 and collagen IV, contribute greatly to the overall stability of the basement membrane.
Junctional epidermolysis bullosa is a skin condition characterized by blister formation within the lamina lucida of the basement membrane zone.
Bromelain, a concentrate of proteolytic enzymes from the pineapple plant, is used in medicine. It is approved in the European Union for the debridement of severe burn wounds under the brand name Nexobrid. It was developed by MediWound.
Scioderm, acquired by Amicus Therapeutics in 2015, was a rare disease company focused on developing a treatment for Epidermolysis Bullosa (EB), a rare genetic disease characterized by extremely fragile skin and recurrent blister formation. There are currently no approved therapies for EB. Scioderm was developing a topical treatment known as SD-101, or Zorblisa, aimed at triggering wound reduction and closure, and a reduction in body surface area coverage of blisters and lesions.
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Beremagene geperpavec, sold under the brand name Vyjuvek, is a gene therapy for the treatment of wounds. Beremagene geperpavec is the first approved gene therapy to use herpes-simplex virus type 1 as a vector. Beremagene geperpavec is a genetically modified herpes-simplex virus used to deliver normal copies of the COL7A1 gene to the wounds.