FDA Grants Castle Creek Rare Pediatric Disease Designation for EB Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Castle Creek Pharmaceuticals’ CCP-020 for the treatment of epidermolysis bullosa simplex, a rare genetic condition that leads to extremely fragile skin.

Epidermolysis bullosa simplex is the most common form of epidermolysis bullosa, which can cause 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.

CCP-020 is a slow-acting compound with potent anti-inflammatory properties. It is delivered in a topical ointment that is active in the epidermis and dermis 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 mid-stage clinical trial that included 17 patients with EBS, 60 percent of patients treated with a cream formulation of CCP-020 experienced at least a 40 percent reduction in blistering after four weeks of treatment, compared to 18 percent treated with a cream without the drug. In this study, adverse events occurred in six patients on CCP-020 compared to 11 patients on cream without the drug. The most notable adverse events were increase in blistering, pruritus. and skin infection. However, none of the listed events were considered treatment related or involved the treatment area.

“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.

The FDA grants Rare Pediatric Disease designation for diseases that primarily affect children from birth to 18 years old and affect fewer than 200,000 persons in the United States. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

If CCP-020 is approved by the FDA for EBS, the Rare Pediatric Disease designation may enable Castle Creek to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive Priority Review for a future drug approval submission, which would reduce the FDA review time from twelve months to six months.

The vouchers are potentially lucrative because they are transferable. Most recently, Spark Therapeutics sold a priority review voucher at the end of April for $110 million.

May 16, 2018
Photo: Michael Derby, co-founder and chief executive officer at Castle Creek Pharmaceuticals