Research & Development
Castle Creek wins US FDA's rare pediatric disease designation for diacerein 1% ointment for epidermolysis bullosa
17 May 2018 -

Transformative therapies company Castle Creek Pharmaceuticals (CCP) disclosed on Wednesday that it has been awarded the US Food and Drug Administration's (FDA) rare pediatric disease designation for diacerein 1% ointment (CCP-020) for the treatment of epidermolysis bullosa (EB).

In conjunction, 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, a rare genetic condition that leads to extremely fragile skin resulting in mild to severe blistering, skin erosion and peeling of the epidermis layers in response to minor injury. There are currently no approved treatment options for any form of EB.

The company said diacerein is a slow-acting, small molecular weight compound of the class anthraquinone with potent anti-inflammatory properties. CCP-020 is a topical formulation of diacerein, 1% ointment. Diacerein in the formulation is hydrolyzed to rhein in the epidermis and dermis following administration and blocks an inflammatory signaling pathway associated with EBS resulting in strengthening epidermal tissue and healing of skin blisters.

Under the company's prior Phase 2 clinical trial in 17 patients with EBS, 60% of patients treated with diacerein cream formulation experienced 40% reduction in blistering after four weeks of treatment, compared to 18% on vehicle. Adverse events occurred in six patients on diacerein versus 11 patients on vehicle.