FDA Grants Fast Track Designation to Castle Creek Pharmaceuticals EBS Treatment

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation to Castle Creek Pharmaceuticals’ CCP-020, its experimental treatment for epidermolysis bullosa simplex.

EB is 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. EBS is a subtype of EB.

CCP-020 is a formulation of diacerein, 1 percent ointment developed for topical application. Diacerein and its active metabolite rhein have been shown to inhibit the production of pro-inflammatory cytokines. Diacerein in the formulation is hydrolyzed to rhein in the epidermis and dermis following administration and is believed to block an inflammatory signaling pathway associated with EBS resulting in strengthening epidermal tissue and healing of skin blisters.

In a previously completed phase 2 clinical trial that included 17 patients with EBS, 60 percent of patients treated with a diacerein cream formulation experienced at least a 40 percent reduction in blistering after four weeks of treatment, compared to 18 percent on placebo. In this study, adverse events occurred in 6 patients on diacerein versus 11 patients on vehicle. The most notable adverse events were increase in blistering (1), pruritus (1) and skin infection (1). However, none of the listed adverse events were considered treatment related or involved the treatment area.

Under the Fast Track designation, Castle Creek Pharmaceuticals, LLC. has the opportunity for priority review and more frequent interactions with the FDA throughout the CCP-020 development program. The FDA previously granted Orphan Drug designation and Rare Pediatric Disease eligibility to CCP-020 for the treatment of epidermolysis bullosa.

“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,” said Amir Tavakkol, executive vice president and chief development officer at Castle Creek Pharmaceuticals. “Fast Track designation for CCP-020 brings new momentum to our goal to bring an effective treatment to people living with EBS in the U.S. and around the world.”

August 9, 2018
Photo: Amir Tavakkol, executive vice president and chief development officer at Castle Creek Pharmaceuticals