Diacerein 1% Ointment for Epidermolysis Bullosa Receives Fast Track Designation

Castle Creek Pharmaceuticals’ diacerein 1% ointment (CCP-020) for the treatment of epidermolysis bullosa simplex (EBS) has received a fast track designation from the US Food and Drug Administration (FDA).

"Fast Track designation is an important milestone in our development program for CCP-020 and reinforces the critical unmet need for patients who are living with the risk of severe blistering and skin erosion associated with EBS," Amir Tavakkol, PhD, executive vice president and chief development officer at Castle Creek Pharmaceuticals, LLC, commented in a recent statement. "With no approved treatment available, patients may be unable to participate in many daily activities and often experience severe pain. Fast Track designation for CCP-020 brings new momentum to our goal to bring an effective treatment to people living with EBS in the United States and around the world."

In a previously completed phase 2 clinical trial, 17 patients with EBS treated with the diacerein cream formulation displayed at least a 40% reduction in blistering after four weeks of treatment, compared to 18% on vehicle in 60% of patients. In 6 patients on diacerein, adverse events (AE) occurred as compared to 11 patients on vehicle. Blistering (1), pruritus (1) and skin infection (1) included the most notable AEs; however, none involved the treatment area or were considered treatment related.

“Based on the data that has been published thus far, CCP-020 shows great promise as a therapy to reduce the severity of blistering in patients with certain forms of EB Simplex," said Anna L. Bruckner, MD, pediatric dermatologist at Children’s Hospital Colorado to Rare Disease Report^®. "Blistering in EBS impacts patients by causing pain, itch, and limitations in activities of daily living, including walking. In addition, the visual nature of the lesions may be stigmatizing and can lead to social isolation. A therapy that reduces the severity of blistering has the potential to reduce the impact of these symptoms on patients, thereby lessening the burden of this chronic disorder.”

Developed for topical application, CCP-020 is a formulation of diacerein, 1% ointment. Following administration, diacerein reins in the epidermis and dermis and works to block a key inflammatory signaling pathway associated with the disease. By doing this, it helps strengthen the epidermal tissue and helps heal skin blisters.

Previously, CCP-020 was granted a rare pediatric disease designation by the FDA.

EBS 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, there are no approved treatments for any form of epidermolysis bullosa.