Galderma Laboratories Trifarotene Development Program Meets Key Milestone For Lamellar Ichthyosis Orphan Disease

LAUSANNE, Switzerland--(BUSINESS WIRE)--Galderma today announced that its trifarotene development program targeting lamellar ichthyosis, a debilitating orphan disease resulting in the formation of scales covering the skin, was demonstrated to be safe and well-tolerated in a phase I study. Trifarotene is a first-in-class molecule being developed by Galderma, the medical solutions business of Nestlé Skin Health.

“Lamellar ichthyosis is a life-long condition significantly affecting the patients’ quality-of-life, and for which there is a great need of effective, safe and specific treatments. Trifarotene with its disease-adapted topical formulation could be a valuable and safe treatment option for patients suffering from this severe congenital disease”

“We are pleased to see the results of this study support the continued development of trifarotene which has the potential to provide a new solution to the needs of patients suffering from lamellar ichthyosis,” says Thibaud Portal, Ph.D., Vice President of Galderma's prescription business. “We are dedicated to meeting the needs of all patients with skin diseases over the course of their lives and trifarotene has been designed to meet the safety profile that patients with lamellar ichtyosis need.”

“Lamellar ichthyosis is a life-long condition significantly affecting the patients’ quality-of-life, and for which there is a great need of effective, safe and specific treatments. Trifarotene with its disease-adapted topical formulation could be a valuable and safe treatment option for patients suffering from this severe congenital disease", said Professor Alain Taieb from Hôpital Saint-Andre in Bordeaux, France and member of the Phase I study board.

The company’s molecule trifarotene is a new retinoic acid receptor (RAR) agonist specifically designed for high selectivity to its target (RAR?) and low systemic metabolic stability, which is currently in clinical development for use in other more common dermatological conditions. Its retinoid functionality and potent keratolytic properties make it a potentially viable treatment of the lamellar ichthyosis pathology. A cream formulation designed to adapt to the disease condition was developed by Galderma, and initial observations in healthy volunteers suggest that the good tolerability of selected dose regimens allow the development of this drug for the treatment of lamellar ichthyosis in future Phase II study.

In 2014, the U.S. Food and Drug Administration granted Orphan Drug Designation for trifarotene in the treatment of congenital ichthyosis. Ichthyoses comprise a large group of skin scaling disorders with diverse etiologies. The stereotypic pathophysiology is epidermal hyperplasia and abnormal desquamation, leading to visible accumulation of squames (scales) on the skin’s surface. Congenital ichthyosis is a term used to refer to a specific group of rare inherited forms of ichthyoses that are generally more severe than non-inherited forms of the disease. Lamellar ichthyosis is one such disorder that falls within the congenital ichthyosis category.

Lamellar ichthyosis is recognised as a severe disease which persists throughout life. After birth, during the first post-natal weeks, the hyperkeratotic (collodion) membrane patients are typically born with is gradually shed and 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, severe discomfort, and significant psycho-social impact. The estimated prevalence of lamellar ichthyosis in the U.S. is in the range of 1 per 100,000 to 1 per 200,000 persons.

In addition to studying treatments for lamellar ichthyosis and other congenital ichthyoses, Galderma is also exploring the treatment of other rare skin diseases such as Cutaneous T Cell Lymphoma and Gorlin Syndrome.

About Galderma

Dating back to 1961, Galderma is now present in over 100 countries with an extensive product portfolio to treat a range of dermatological conditions. The company partners with health care professionals around the world to meet the skin health needs of people throughout their lifetime. Galderma is a leader in research and development of scientifically-defined and medically-proven solutions for the skin, hair and nails.