The FDA has granted rare pediatric disease designation for Castle Creek Pharmaceuticals' (CCP) diacerein 1% ointment (CCP-020) for the treatment of epidermolysis bullosa (EB). The safety and efficacy of CCP-020 is currently being evaluated in the DELIVERS study for the treatment of patients with epidermolysis bullosa simplex (EBS), a subtype of EB.
"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," said Michael Derby, co-founder and chief executive officer at Castle Creek Pharmaceuticals. "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."
Rare pediatric disease designation indicates that the FDA may give the company a priority review voucher if the drug is approved for the rare pediatric indication. A rare pediatric disease designation is given to a drug candidate that has demonstrated the potential to offer significant improvement in safety and efficacy for a serious or life-threatening rare disease.