Galderma's Skin Disease Drug Granted Orphan Drug Designation

Galderma has announced that the FDA has granted the company’s trifarotene molecule Orphan Drug Designation status.

The company has stated that the news of the FDA decision means Galderma can continue to focus on developing clinical strategies for treating all patients with skin diseases. Humberto Antunes, the president and CEO of Galderma, commented on the company’s process. “Given the burden of congenital ichthyosis and the lack of effective and easy-to-use treatments, this is a condition which is very difficult to live with. Galderma’s objective is therefore not only to provide an efficient and safe medical solution to alleviate the symptoms of the disease, but also to improve patients’ quality of life,” said Antunes.

The trifarotene molecule is a selective agonist of the gamma retinoic acid receptor (RAR-gamma). The molecule is being used for the treatment of congenital ichthyosis and being researched as a potential treatment for a number of other more common dermatological conditions. Congential ichthyosis refers to a specific group of rare and inherited forms of ichthyoses, which are skin scaling disorders. One severe ichthyosis, lamella ichthyosis, causes scaling and lichenification that covers the entire body. Lamellar ichthyosis can result in severe disability, such as partial deafness, discomfort, and psycho-social issues. 

Galderma is also exploring the treatment of rare diseases, such as Cutaneous T Cell Lymphoma and Gorlin Syndrome. Galderma has noted that it continues to work with regulatory agencies in order to make the clinical process for the trifarotene molecule as expedient as possible.

The orphan drug designation will speed up the clinical process, as the FDA uses it as an incentive for drug companies to produce rare drugs. If Galderma’s drug makes it to market, the company will have a longer period of exclusive marketing.