Rare Disease Report

Topical Diacerein Showing Promise for Epidermolysis Bullosa

OCTOBER 02, 2016
Andrew Black
Good news for epidermolysis bullosa (EB) patients.  Castle Creek Pharmaceuticals presented exciting new Phase 2 data at the 25th Annual Congress of the European Academy of Dermatology and Venereology (EADV) in Vienna showing topical diacerein 1% to be effective in some patients.
The Phase 2 trial was a multicenter, randomized, double-blind, placebo-controlled study assessing a 4-week treatment of topical diacerein 1% in 17 patients ages 4-12 years-old with a K14 or K5 gene mutations of EB. 
After 4 weeks, patients then followed a 12-week follow up phase in 2 years with a crossover after the first year.
The primary objective of the trial was reduction of blister numbers by 40% in the treated skin area (3% of body surface) of the patients compared with placebo after 4 weeks.


60% of patients who received topical diacerein 1% achieved the primary end point with at least a 40% reduction in the number of blisters at 4 weeks. Only 15% of placebo patients showed a similar benefit.
Continued benefit of topical diacerein 1% was also demonstrated after stopping the treatment through the follow up period of 12 weeks.
Treatment was well tolerated by patients with no related adverse events reported.

About Diacerin

Diacerein is the prodrug of rhein, a small molecule that blocks the activity of Interleukin-1β (IL-1β) by inhibiting plasma membrane-bound IL-1 converting enzyme3. IL-1β is a pro-inflammatory cytokine that has been demonstrated to be upregulated in EB.

About Epidermoylsis Bullosa 

Epidermolysis bullosa is a group of rare genetic rare disorders that manifest as blistering or erosion of the skin. There are several types of EB and in the more severe forms of the disease, blistering can lead to deformities including fusion of the fingers and toes, secondary skin infections, sepsis and even death.  The deformities, constant pain, and rarity of the condition can make the disease a very isolating one.  

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