Abstract: Congenital aniridia is a rare and severe genetic panocular disease characterized by a complete or partial iris defect clinically detectable at birth. The most common form of aniridia occurring in around 90% cases is caused by PAX6 haploinsufficiency. Ataluren eye-drops aim to restore ocular surface PAX6 haploinsufficiency in Aniridia Related Keratopathy (ARK). However, they are currently no ophthalmic solution available forms. The objective of this study was to assess the physicochemical and microbiological stability of ataluren eye-drop 1% in preservative-free low-density polyethylene (LDPE) bottle with an innovative insert that maintains sterility after opening. Because ataluren is a strongly lipophilic compound, the formulation is complex and involves a strategy based on co-solvents in an aqueous phase or an oily formulation capable of totally dissolving the active ingredient.

Abstract: A topical composition including erlotinib and a pharmaceutically acceptable excipient for use in the treatment of a keratoderma in a child, preferably a palmoplantar keratoderma (PPK), wherein the composition is topically administered. The composition may further include a penetration enhancer. The treated subject may be younger than three years old. Also, woven or non-woven fabric support including erlotinib and to dressings, patches, gloves and socks having the support useful in the treatment of a PPK.