Prademagene zamikeracel promotes wound healing in rare genetic skin disease

In patients with large chronic wounds secondary to recessive dystrophic epidermolysis bullosa (RDEB), treatment with prademagene zamikeracel helps with wound healing and pain while being well tolerated, according to a phase III study.

The study included 11 paediatric and adult patients (median age 21 years, 64 percent female) with RDEB who at least two chronic wounds greater than 20 cm², had no evidence of an immune response to type VII collagen, and expressed the amino-terminal NC1 fragment of type VII collagen.

A total of 86 wounds were matched and randomized to treatment with prademagene zamikera (n=43) or to standard of care (control; n=43). Patients in the active treatment group underwent a surgical procedure, wherein prademagene zamikeracel, an autologous COL7A1 gene-modified cellular sheet, was sutured onto the wound. A maximum of six wounds could be treated with prademagene zamikeracel per patient.

The coprimary endpoints were the proportion of wounds with at least 50-percent healing and pain reduction from baseline at week 24. Safety was also assessed.

At week 24, 81 of wounds treated with prademagene zamikeracel were at least 50 percent healed as opposed to 16 percent of wounds treated with standard of care (mean difference, 67 percent, 95 percent confidence interval [CI], 50–89; p<0.0001).

Wound pain scores decreased by 3.07 points with prademagene zamikeracel and by only 0.90 points with standard of care (mean pairwise difference, –2.23, 95 percent CI, –3.45 to –0.66; p=0.0002).

None of the patients experienced serious treatment-related adverse events.