FDA grants orphan drug status for congenital ichthyosis treatment

July 8, 2014

The Food and Drug Administration has granted orphan drug designation to Galderma’s trifarotene molecule for the treatment of congenital ichthyosis.

The Food and Drug Administration has granted orphan drug designation to Galderma’s trifarotene molecule for the treatment of congenital ichthyosis.

As a result, Galderma officials say the company plans to implement a clinical development plan to explore new treatment options for other rare skin diseases such as cutaneous T-cell lymphoma and Gorlin syndrome.

The orphan drug designation is reserved for drugs and biologics that are intended for the effective treatment, diagnosis or prevention of rare disorders that affect fewer than 200,000 people in the United States or that affect more than 200,000 people but are not expected to generate enough revenue to cover development and marketing costs.

“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 President and CEO Humberto C. Antunes said in a news release. “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 trifarotene molecule is “a selective agonist of the gamma retinoic acid receptor (RAR-gamma),” which is currently in clinical development for use in more common dermatological conditions, according to the company. The drug’s retinoid functionality and keratolytic properties make it a potentially viable treatment for lamellar ichthyosis.

Galderma has initiated the program for investigating the treatment of lamellar ichthyosis with trifarotene and is currently working in collaboration with regulators to implement a clinical development plan.

The estimated domestic prevalence of lamellar ichthyosis is in the range of one per 150,000 persons.