Amarantus BioScience Holdings, Inc., announced it has requested Rare Pediatric Disease Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) to use its Engineered Skin Substitute (ESS) as a potential treatment for a childhood skin disease known as giant congenital melanocytic nevus (GCMN).
GCMN is a dermatological condition found in between 8 and 80 babies born annually in the United States, and, under the FDA’s definition of rare diseases, presently affects less than 200,000 people. The disease, also known by names that include “Bathing trunk nevus,” “Garment nevus,” or “Giant hairy nevus,” is distinguished by large, darkly pigmented and often hairy patches, which can vary in consistency and number, on any part of the body. One of this condition’s serious risks is the potential to develop melanoma, generally within the first 10 years of life. Another is neurocutaneous melanocytosis (NCM), a life-threatening neurological and dermatological illness marked by the abnormal cellular nodes within the central nervous system and the skin.
Amarantus’ ESS is a tissue-like skin, made from the patient’s own skin cells. It combines cultured epithelium with a collagen-fibroblast implant to produce a skin-like tissue with both epidermal and dermal components, and has been observed in preclinical studies to generate a functional skin barrier. Importantly, because ESS consists of the patient’s own cells, it is less likely to be rejected by the immune system than porcine or cadaver grafts.
The company reports that ESS has already been used in over 130 people, primarily pediatric patients in investigator-initiated clinical settings, to treat severe burns affecting more than 95% of the body’s surface.
Treatments approved through the FDA’s Rare Pediatric Disease Priority Review Voucher program are eligible for vouchers that may be redeemed to obtain expedited FDA review for subsequent marketing applications.
The FDA’s Orphan Drug Designation, if granted, can offer drugs and biologics intended to treat, diagnose or prevent rare diseases seven years of marketing exclusivity against competition, plus incentives that may include federal grants, tax credits, and filing fee waiver.