PARSIPPANY, N.J., May 16, 2018 – Castle Creek Pharmaceuticals (CCP), a global company dedicated to delivering transformative therapies to patients with orphan dermatologic and other underserved conditions, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for 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.
EBS is the most common form 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.
Diacerein is a slow-acting, small molecular weight compound of the class anthraquinone with potent anti-inflammatory properties. Diacerein and its active metabolite rhein have been shown to inhibit the in vitro and in vivo production and activity of IL-1β and other pro-inflammatory cytokines. CCP-020 is a formulation of diacerein, 1% ointment developed for topical application. 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% of patients treated with a diacerein cream formulation experienced at least a 40% reduction in blistering after four weeks of treatment, compared to 18% on vehicle.[i],[ii] In this study, adverse events (AE) occurred in 6 patients on diacerein versus 11 patients on vehicle. The most notable AEs were increase in blistering (1), pruritus (1) and skin infection (1). However, none of the listed events were considered treatment related or involved the treatment area.
About Castle Creek Pharmaceuticals
Castle Creek Pharmaceuticals (CCP), part of the Paragon Biosciences portfolio of innovative biotech companies, is a privately held biopharmaceutical company developing innovative therapies for patients with rare, serious or debilitating dermatologic conditions. The company has a robust and diversified pipeline of late-stage products that have the potential to transform lives. For more information, please visit www.castlecreekpharma.com.
[i] Wally et. al., Diacerein for the treatment of epidermolysis bullosa – a phase II randomized, placebo controlled, double-blind multi-center clinical trial, 25th EADV congress, Vienna, Austria 2016.
[ii] Total (%) patients with >40% reduction of blister numbers after two 4-week intervention with diacerein or its vehicle for all 17 patients during a 2-year cross-over trial.