Safety, tolerability, and efficacy of a novel topical isotretinoin formulation for the treatment of X-linked or lamellar congenital ichthyosis: Results from a phase 2a proof-of-concept study

Abstract

To the Editor: Clinical characteristics of congenital ichthyosis (CI) include hyperkeratosis, scaling, and erythroderma.1,2 The management of CI typically involves emollients and keratolytics.2,3 Patients with lamellar or X-linked recessive CI often have considerable hyperkeratosis and scaling, for which systemic retinoids are effective but may cause dose-limiting adverse events (AEs).2,4,5 PAT-001 is a patented, novel, topical isotretinoin ointment formulation to treat CI. We present the results from a phase 2a study evaluating PAT-001 in patients with lamellar or X-linked recessive CI.