Topical isotretinoin shows early efficacy in congenital ichthyosis

March 09, 2022

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Source/Disclosures


Disclosures: Paller reports financial relationships with AbbVie, Abeona, Almirall, Amagma, AnaptysBio, Arena, Bausch, Bristol Myers Squibb, Dermavant Sciences, Dermira, Eli Lilly, Exicure, Forte, Galderma, Incyte, Krystal Biotech, LEO Pharma, LifeMax, Janssen, Novartis, Pierre Fabre, Pfizer, Rapt, Regeneron, Sanofi, Sol30 Gel, UCB and Venthera. Please see the study for relevant financial information from all other authors.


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A new topical formulation of isotretinoin was safe and effective in the treatment of congenital ichthyosis, according to a proof-of-concept study.

“Clinical features of ichthyosis congenita (IC) include hyperkeratosis, desquamation, and erythroderma”, Amy S. Paller, MD, Professor Walter J. Hamlin and chair of dermatology at Northwestern University Feinberg School of Medicine and pediatric dermatologist at the Ann and Robert H. Lurie Children’s Hospital in Chicago, and his colleagues wrote in a research letter. “Management usually involves emollients and keratolytics. Patients with lamellar (LI) or X-linked recessive (XLRI) IC often present with considerable hyperkeratosis and desquamation for which systemic retinoids are effective but may cause dose-limiting adverse events (AEs).

Multicenter, double-blind, Phase 2a study evaluated PAT-001 0.1% or 0.2% (Patagonia Pharmaceuticals), a novel topical isotretinoin ointment, in pediatric patients with HF relative to the vehicle.

Ten subjects were enrolled in the 0.1% group and nine in the 0.2% group, of whom seven had XLRI, 10 had LI and two had ichthyosis vulgaris.

AEs were reported in 14 patients, most of them mild. AEs were ‘possibly’ treatment-related in 25% of patients, ‘probably’ treatment-related in 3.6% and ‘definitely’ treatment-related in 10.7%. Seven patients stopped treatment.

An improvement of at least 1 grade in the Investigator’s Global Assessment score was reported in 100% of people in the 0.1% group compared to 66.7% in the placebo group, while an improvement in 2 or more grades was reported in 66.7% and 33.3%, respectively. .

In the 0.2% group, IGA improvement of 1 grade or greater was reported in 100% of treated individuals compared to 87.5% in the vehicle group, while IGA improvement of Grade 2 or higher was reported in 50% of treated individuals compared to 62.5% of the vehicle group.

Desquamation was improved to clear, almost clear or slight in 100% of both treatment groups and 55.6% and 87.5% of each vehicle group.

“The results indicate no concerning safety signals or evidence of systemic exposure to isotretinoin/tretinoin,” the authors wrote. “PAT-001 0.1% seemed to have a stronger efficacy signal than 0.2%.”

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