The FDA granted orphan drug designation status for Galderma's trifarotene molecule for the treatment of congenital ichthyosis. Based on this decision, Galderma says it plans to implement a clinical development plan, reinforcing its commitment to exploring new treatment options for rare diseases, as well as meeting the needs of all patients with skin diseases over the course of their lives.

“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,” says Humberto C. Antunes, Galderma President & CEO.

Trifarotene is a selective agonist of the gamma retinoic acid receptor (RARγ), which is currently in clinical development for use in other more common dermatological conditions. It is the drug's retinoid functionality and potent keratolytic properties that make it a potentially viable treatment of the lamellar ichthyosis pathology, according to the company. Galderma has already initiated the program for investigating the treatment of lamellar ichthyosis with trifarotene and is currently working in collaboration with regulatory authorities to implement an innovative and expedient clinical development plan.

Lamellar ichthyosis, which falls within the congenital ichthyosis category, is recognized as a severe disease that persists throughout life. After birth, during the first post-natal weeks, the hyperkeratotic (colloidion) membrane patients are typically born with, is gradually shed and is replaced by scaling and lichenification that involves the entire body, including face, scalp, palms and soles. While usually not life threatening, lamellar ichthyosis can result in disability, partial deafness, poor adaptation to environmental conditions (due to hypohydrosis), severe discomfort (pruritus, fissuring of the skin), and significant psycho-social impact. The estimated prevalence of LI in the US is in the range of 1 per 100,000 to 1 per 200,000 persons.