Rare Disease Breakthrough? New Treatment May Reduce Blistering in EBS

In a possible breakthrough in the treatment of epidermolysis bullosa simplex (EBS), Diacerein 1% ointment reduced blistering in patients with this rare genetic connective tissue disorder.

The new findings were presented in a late-breaker session at the American Academy of Dermatology Annual Meeting in Orlando.

There are currently no approved treatment options for EBS, which is the most common form of epidermolysis bullosa. The investigational diacerein 1% ointment (CCP-020), under development by Castle Creek Pharmaceuticals, blocks an inflammatory signaling pathway.

The multicenter, randomized, double-blind, placebo-controlled phase 2 trial included 17 patients with EBS who were treated for four weeks followed by three-month follow-up and subsequent cross-over in year two. Results showed a 60 percent reduction in blistering among patients treated with diacerein 1% versus a 15 percent reduction in the placebo group at four weeks. At three months, 67 percent of patients in the placebo group returned to baseline blistering levels, versus 12.5 percent of patients in the diacerein 1% treatment group. Topical diacerein 1% was well tolerated with no treatment-related adverse events reported.

“With no treatment options available, management of EBS remains a significant area of unmet need in healthcare. These results represent a historic advance in research related to EBS indicating the potential to bring patients a safe and effective treatment option in the years ahead,” says study author Johann Bauer, MD, MBA, HCM, head of the University Clinic for Dermatology at the SALK/Paracelsus Medical University in Salzburg, Austria, in a news release. “Importantly, these results also show the potential for diacerein 1% to offer long-term benefits in patients experiencing blistering associated with EBS.”