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Orphan Drug From Scioderm Aims to Treat Rare Skin Disease

Xconomy Raleigh-Durham — 

For children with epidermolysis bullosa, the debate about whether to pull off Band-Aids quickly or peel them back slowly does not apply. From head to toe, special bandages cover skin so fragile that anything but the most slow and careful removal can rip open a wound worse than the one the dressing was protecting. Brett Kopelan, executive director of the Dystrophic Epidermolysis Bullosa Research Association of America (DebRA), and the parent of a 7-year-old girl who has the disease, says he has seen children rip their own skin scratching itchy wounds.

There are no approved drugs that treat epidermolysis bullosa or heal its chronic wounds. But Durham, NC company Scioderm is on the cusp of late-stage clinical studies for an experimental skin cream that, if proven effective in improving wound healing, could become the first Food and Drug Administration-approved treatment for the disease.

“If they’re right, it would be a huge quality of life impact,” Kopelan says of Scioderm’s efforts. “Just the idea of being able to wear shoes more regularly, or (use) less bandages that are constricting, from a pain point of view or an itch point of view it would be almost a game change.”

Epidermolysis bullosa, or EB, is a group of connective tissue disorders caused by gene mutations in any one of 18 genes that may block or disrupt the formation of collagen, a structural protein that attaches the skin’s layers. Without collagen to connect the epidermis to the underlying dermis, even a slight chafing can blister or wound the skin. EB patients are sometimes called “butterfly children” because their skin is delicate, like a butterfly’s wings. DebRA counts 30,000 EB cases in the United States and 500,000 worldwide.

There are four types genetically-inherited EB; the disease can range from mild to devastatingly severe. Babies with the most severe forms usually die in their first year—mostly from infections in open wounds. Those who live into the teenage years and older face the additional risk of developing an aggressive form of skin cancer. Rarely does anyone with the more severe forms of the disorder live past the age of 30.

Scioderm’s experimental treatment is a topical cream (named Zorblisa) containing the active ingredient allantoin. Allantoin is a natural substance found in some plants and the urine of many mammals, and that has found use in dermatological products. It is known to soften keratin, the fibrous proteins in skin, enabling skin to hold more moisture. It’s already FDA approved as a cosmetic ingredient used in over-the-counter items like toothpaste, shampoo, and even some skin care products. But allantoin is unstable and has a short half life, Kopelan says. Scioderm’s cream keeps allantoin stable and delivers it in a higher concentration than commercially available skin products.

The cream did not originate with Scioderm. It was first tested in a 2009 clinical trial by the now defunct Minnesota skin products firm, Alwyn Company, Inc. Robert Ryan, Scioderm’s CEO and co-founder, says families of EB patients had asked Alwyn for a Vaseline-like barrier cream to prevent bandages from sticking to skin. Alwyn developed a topical cream that contained allantoin. Even through the cream had a low concentration of the ingredient, patients reported … Next Page »

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