Rare Disease Report

FDA Grants Rare Pediatric Disease Designation to EBS Treatment

MAY 16, 2018
Krista Rossi
This morning, May 16, 2018,  the US Food and Drug administration (FDA) granted rare pediatric disease designation to Castle Creek Pharmaceuticals (CCP) for diacerein 1% ointment (CCP-020) for the treatment of epidermolysis bullosa simplex (EBS). The drug’s safety and efficacy are being evaluated at present in the DELIVERS study.

EBS is the most common form of epidermolysis bullosa. It is a rare, genetic skin condition in which the skin is extremely fragile, oftentimes resulting in severe blistering, skin erosion, and peeling of the epidermis layers in response to minor injuries and traumas.

Currently, no approved treatments exist for any form of epidermolysis bullosa.

CCP-020 is a formulation of diacerein, 1% ointment developed for topical application. The formulation contains diacerein hydrolyzed to rhein in the epidermis and dermis (post administration). It is believed to block an inflammatory signaling pathway associated with EBS, which results in the healing of skin blisters and strengthening of epidermal tissue.

"Receiving this important rare pediatric disease designation is a significant recognition of CCP-020 and its potential as an important therapy for people living with EBS," stated Michael Derby, co-founder and chief executive officer at Castle Creek Pharmaceuticals in a recent comment. "We look forward to advancing the clinical development of CCP-020 in our ongoing DELIVERS study and fulfilling our mission to improve the health and quality of life for people living with EBS in the years ahead."

Seventeen patients in a previously completed phase 2 clinical trial experienced positive outcomes with CCP-020. Sixty percent of patients experienced a minimum of a 40% reduction in blistering after 4 weeks of treatment with the diacerein cream formulation as compared with 18% on the vehicle. Six patients on diacerein experienced adverse events (AEs) in comparison with 11 patients on the vehicle. Blistering, pruritus, and skin infection were the most prominent AEs. However, they were not considered treatment related, and they did not involve the treatment area.

Secondary outcome measures included the efficacy of diacerein 1% ointment compared with control ointment; the efficacy of diacerein 1% ointment compared with control ointment based on reduction in the body surface area (BSA) of EBS lesions; pain intensity as assessed using the Numeric Rating Scale; pruritus intensity as assessed using the Numeric Rating Scale; a mobility assessment using a series of age-appropriate questions in an eDiary; and the number of patients with adverse events based on assessment of safety measures.

The estimated completion date for the study is November 2018.

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