Galderma Granted FDA Orphan Drug Status For Rare Skin Disease Drug

The U.S. Food and Drug Administration (FDA) has bestowed Orphan Drug Designation to Galderma’s trifarotene molecule for the treatment of the skin disease congenital ichthyosis.

Galderma’s molecule trifarotene is a selective agonist of the gamma retinoic acid receptor (RARγ). The lead molecule, which possesses retinoid functionality and potent keratolytic properties is currently under investigation for the treatment of lamellar ichthyosis pathology. Galderma said that a clinical development program for the molecule is in the works and it is currently interacting with regulatory authorities for its implementation.

Congenital ichthyosis is an umbrella term for a group of rare, inherited forms of ichthyoses, a large group of skin scaling disorders. Lamellar ichthyosis is one of the disorders that belong to the congenital ichthyosis category. The disease manifests during the first weeks of life and lasts throughout a patient’s lifetime. During the first post-natal weeks, the infant’s hyperkeratotic membrane is shed and replaced by scaling and lichenification that covers the entire body. The disorder, while not typically life-threatening, can lead to disability, partial deafness, severe discomfort, and psycho-social impacts. Lamellar ichthyosis affects one per 100,000 to one per 200,000 in the U.S.

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

With the orphan drug designation, the company said it plans to conduct a clinical development plan to explore novel treatments for rare diseases as well as unmet medical needs of patients with skin diseases. Orphan status confers a range of benefits for the sponsor company, including a seven year period of U.S. marketing exclusivity upon approval and financial incentives.