Skinvisible, Inc. announced the submission of an application for an Orphan Drug Product Designation for the company's product formulated with Invisicare to treat Netherton Syndrome, a sub-type of ichthyosis disease. In conjunction with this application, Skinvisible has also filed a patent application with the US Patent Office.
Netherton Syndrome is a sub-type of ichthyosis disease which is also referred to as NETH, Comel-Netherton syndrome, ichthyosis linearis circumflexa, or trichorrhexis invaginata. Netherton Syndrome is a genetic disease that is characterized by excessively scaly, circular red skin, brittle hair and for some also atopic dermatitis. The disease, which presents itself at birth or within the first year, is a life-long affliction that can have debilitating and even fatal consequences. There are currently no treatments available for Netherton Syndrome and limited options to treat the symptoms.
Patients with Netherton Syndrome have a mutated SPINK5 gene which is the gene responsible for inhibiting serine proteinase. The role of serine proteinase is to breakdown the intra-cellular bounds of the skin. Without the inhibitor, the breakdown of skin is no longer repressed, resulting in chronic scaly, red skin typical of Netherton syndrome. Skinvisible's formulation is a serine proteinase inhibitor which is applied topically to the skin to prevent this constant breakdown of the skin.
The United States Orphan Drug Act was created to provide incentives to pharmaceutical companies to develop products for patients suffering from rare diseases. In the United States, a rare disease is defined as a condition which has less than 200,000 people with the disease. The incentives stipulated by the Act include eligibility for seven years of market exclusively for the product as well as a waiver of fees, tax incentives and a potential for grants to fund clinical trials. Some orphan drugs also receive an expedited review if the disease is severe or life-threatening.
"Receiving an orphan drug designation would provide exclusive marketing rights for this product in the United States, which is a key benefit to Skinvisible and potential licensees of this product," said Terry Howlett, president and CEO of Skinvisible. "Our next objective will be to find a partner to take this product through the clinical path to FDA approval and then leverage the results of that process for more rapid expansion into other diseases."