Galderma announced today that it received U.S. FDA Orphan Drug Designation status for its trifarotene molecule, a selective agonist of the gamma retinoic acid receptor (RARγ), for the treatment of congenital ichthyosis.
Now, the company says it is working to implement a clinical development plan exploring new treatment options for rare skin diseases.
The drug, which is also in clinical development for more common skin conditions, is said to be a potentially viable treatment of the lamellar ichthyosis (LI) pathology because of its retinoid functionality and potent keratolytic properties. Galderma has begun working on an investigational program for LI with trifarotene and has partnered with regulators to implement a clinical development plan, according to the company’s announcement.
Ichthyoses is made up of a large group of skin scaling disorders with diverse etiologies, according to Galderma. While the disease is normally not life threatening, the LI strain has been known to cause disability, partial deafness, poor adaptation to environmental conditions, and significant psycho-social impact. It’s estimated that the 1 in about 200 thousand people suffer from LI in the U.S.
“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,” Humberto C. Antunes, Galderma president & CEO, said. “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.”
The Orphan Drug Designation is given to drugs and biologics which are “defined as those intended for the safe and , effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing,” Galderma explained.
The company added that it is also exploring the treatment of other rare skin diseases such as Cutaneous T Cell Lymphoma and Gorlin Syndrome.