FDA Grants Rare Pediatric Disease Designation for CCP-020 for Treatment of Epidermolysis Bullosa

The US Food and Drug Administration (FDA) granted rare pediatric disease designation for CCP-020 (diacerein 1% ointment) for treatment of epidermolysis bullosa, announced Castle Creek Pharmaceuticals.

The safety and efficacy of the ointment is currently being evaluated in the DELIVERS study for treatment of patients with epidermolysis bullosa simplex (EBS), a subtype of epidermolysis bullosa.

The ointment is a potentially disease-modifying investigational drug that may block an important inflammatory signaling pathway associated with EBS.

“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,” Michael Derby, co-founder, chief executive officer, Castle Creek Pharmaceuticals, said in a statement. “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.”

The DELIVERS study, an international phase 2 clinical trial, with clinical research sites in the US, Europe, Israel and Australia, that will include about 80 participants who will be randomized in a double-blind design, in 1 of 2 parallel-groups.

The efficacy of diacerein 1% ointment will be compared to a control ointment when applied once-daily for 8 weeks. Randomization will be stratified by genotype and age group. Participants will report outcomes including pruritus, pain and mobility each day via an electronic diary, and follow-up visits will continue for a maximum of 22 weeks.

The estimated completion date for the study is November 2018.

Diacerein is a slow-acting, small molecular weight compound of the class anthraquinone with potent anti-inflammatory properties.

In the formulation, diacerein is hydrolyzed to its active metabolite rhein in the epidermis and dermis following administration and is believed to block an inflammatory signaling pathway associated with EBS which results in the strengthening of the epidermal tissue and healing of skin blisters.

A phase 2 clinical trial has been completed that included 17 patients with EBS, where 60% treated with a diacerein cream formulation experienced at least a 40% reduction in blistering after 4 weeks of treatment versus 18% on vehicle.

In the study, adverse effects occurred in 6 patients on diacerein versus on vehicle, with the most notable including blistering, pruritus and skin infection, however, none were considered treatment related or involved the treatment area.

There are currently no treatment options available for any form of epidermolysis bullosa aside from supportive care that includes wound care, use of bandages and pain management.