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The Food and Drug Administration (FDA) has approved rare pediatric disease designation for diacerein 1 percent ointment (CPP-020, Castle Creek Pharmaceuticals) for the treatment of epidermolysis bullosa (EB).
The designation means the FDA could give Castle Creek a priority review voucher if diacerein is approved for the rare pediatric indication.
Diacerein contains anti-inflammatory properties and contains an active metabolite, rhein, that has demonstrated inhibition of in vitro and in vivo production and activity of interleukin-1 beta and other inflammatory cytokines. CCP-020 was formulated to block an inflammatory signaling pathway associated with EBS, which helps to heal blisters and strengthen epidermal tissue, according to a news release.
CCP-020’s safety and efficacy are being assessed in the DELIVERS study for patients with epidermolysis bullosa simplex (EBS). A separate, phase 2 clinical trial that has completed involved 17 patients with EBS. Of the study participants, 60 percent demonstrated a 40 percent reduction in blistering after four weeks of treatment with diacerein ointment, compared to 18 percent on vehicle. Notable adverse events included increases in blistering, pruritus and skin infections. None of the adverse events were related to treatment or involving the treatment areas.
Receiving this important rare pediatric disease designation is a signification recognition of CCP-020 and its potential as an important therapy for people living with EBS,” Castle Creek co-founder and CEO Michael Derby said