![]() | A major contributor to this article appears to have a close connection with its subject.(September 2015) |
Industry | Pharmaceuticals |
---|---|
Founded | April 2013 |
Founders | Robert Coull, CA, CBV Robert Ryan, Ph.D. |
Headquarters | |
Products | SD-101 for epidermolysis bullosa (EB) |
Brands | Zorblisa |
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. [1]
Epidermolysis Bullosa, sometimes referred to as "Butterfly Skin", [2] is a rare genetic connective tissue disorder that, in all forms, results in extremely fragile skin that blisters or tears at the slightest friction or trauma. [3] EB typically manifests at birth or early childhood. [4] According to the Dystrophic Epidermolysis Bullosa Research Association of America (DEBRA), an estimated 1 out of every 20,000 live births are affected with some type of EB and the disorder occurs in every racial and ethnic group throughout the world and affects both sexes equally. [5]
In Scioderm's initial open-label Phase 2 study conducted in children with either Simplex, Recessive Dystrophic (RDEB), or Junctional EB, the application of SD-101 resulted in complete closure of 88% of target chronic lesions within one month, in addition to a 57% reduction in Body Surface Area (BSA) coverage of lesions and erosions after 3 months of daily treatment. [6] [7]
SD-101, now owned by Amicus Therapeutics, is currently in Phase 3 clinical development to evaluate Zorblisa as a therapy for the treatment of lesions and blistering associated with Epidermolysis Bullosa. [8] Zorblisa is the first drug to ever enter Phase 3 development for the treatment of EB. [1] Stem cell research for Epidermolysis Bullosa is also underway by researchers at the University of Minnesota pursuant to an open-label Phase 2 trial. [9]
In April 2013 Scioderm received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for its topical treatment, SD-101 for Epidermolysis Bullosa. [7] As a result of receiving Breakthrough therapy designation for SD-101, Scioderm was named a 2013 "Fierce Top 15" company by FierceBiotech. [10] [11] Prior to Scioderm's receipt of the FDA's Breakthrough therapy designation for SD-101, it had only been given to high-profile pharmaceutical companies such as Johnson & Johnson, Merck and Novartis. [12] In addition to Breakthrough therapy designation, Scioderm received Orphan Drug Designation from both the FDA and from the European Medicines Agency (EMA) for its EB treatment. [13]
Scioderm was co-founded by Robert Coull and Robert Ryan. [14] [15] [16] Coull and Ryan "acquired the asset from another firm, which had demonstrated a wound healing effect at a lower concentration of the active ingredient before the topical [treatment's] advancement was stalled by lack of funding." [16] In April 2013, the company received $16 million in Series A Financing from Morgenthaler Ventures and Technology Partners. [17]
On August 31, 2015, Amicus Therapeutics announced the acquisition of Scioderm in a deal valued at approximately $950 million based on the achievement of certain milestones. [18] [19] Amicus plans to complete the clinical development of SD-101 and to make the treatment commercially available for all EB patients as quickly as possible. [18]
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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 the curing of epidermolysis bullosa, with national groups in over 40 countries including in the United Kingdom and the United States.
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Epidermolysis bullosa dystrophica or dystrophic EB (DEB) is an inherited disease affecting the skin and other organs.
Genodermatosis is a hereditary skin disease with three inherited modes including single gene inheritance, multiple gene inheritance and chromosome inheritance. There are many different types of genodermatosis, the prevalence of genodermatosis ranges from 1 per 6000 people to 1 per 500,000 people. Genodermatosis has influence on the texture, color and structure of skin cuticle and connective tissue, specific lesion site and clinical manifestations on the body vary depending on the type. In the spite of the variety and complexity of genodermatosis, there are still some common methods that can help people diagnose. After diagnosis, different types of genodermatosis require different levels of therapy including interventions, nursing interventions and treatments. Among that, research of therapy for some new, complex and rare types are still in the developing stage. The impact of genodermatosis not only can be seen in body but also can be seen in all aspects of patients' life, including but not limited to psychological, family life, economic conditions and social activities. Accordingly, the patients need treatment, support and help in these areas.
Protein replacement therapy is a medical treatment that supplements or replaces a protein in patients in whom that particular protein is deficient or absent. There have been significant advances in this treatment. PRT is being tested in clinical trials with the diseases progeria and epidermolysis bullosa dystrophica as a potential treatment. For patients with epidermolysis bullosa dystrophica there have been promising results.
Junctional epidermolysis bullosa is a skin condition characterized by blister formation within the lamina lucida of the basement membrane zone.
A coma blister, or coma bullae, is a skin lesion or blister that typically arises due to pressure in an individual with impaired consciousness. They vary in size, ranging from 4 to 5 centimeters in diameter, and may appear hemorrhagic or blood filled. Coma blisters are usually found in the extremities and trunk. These types of blisters have been associated with the overdose of central nervous system (CNS) depressants especially barbiturates, but also tricyclic antidepressants, hypnotics, benzodiazepines, opiates, antipsychotics, and alcohol. However, studies have found that coma blisters are not caused by the toxicity of these drugs, but due to hypoxia and external pressure on the comatose individual's skin from being immobilized. Coma blisters have been frequently found on individuals who have overdosed on drugs, but have also been found on individuals with chronic kidney failure, hypercalcemia, diabetic ketoacidosis, and a variety of neurologic conditions. Coma blisters are more frequent in adults and less common among children as demonstrated by the few cases published in literature.
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Emapalumab, sold under the brand name Gamifant, is an anti-interferon-gamma (IFNγ) antibody medication used for the treatment of hemophagocytic lymphohistiocytosis (HLH), which has no cure.
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