The U.S. Food and Drug Administration (FDA) has approved Zevaskyn (prademagene zamikeracel or pz-cel) for the treatment of wounds in patients with recessive dystrophic epidermolysis bullosa (EB).
EB is a group of rare genetic skin diseases that cause the skin to blister and erode very easily. In people with EB, blisters form in response to minor injuries or friction, such as rubbing or scratching. Recessive dystrophic EB is usually caused by changes in the COL7A1 gene.
Pz-cel is the first and only autologous cell sheet-based gene therapy for the treatment of recessive dystrophic EB. The treatment targets defective COL7A1 genes by administering a functional type VII collagen-producing COL7A1 gene.
The FDA’s approval follows positive data from the phase 3, multicenter, randomized, intrapatient-controlled VITAL clinical trial (NCT04227106). The study met its co-primary endpoints by demonstrating statistically significant healing of 50% or greater from baseline in large chronic EB wounds and pain reduction from baseline evaluated at six months post-treatment. Across 43 chronic wounds treated with a single dose of pz-cel, 81% showed 50% or greater healing at six month evaluation, compared to 16% in the control group.
Pz-cel was observed to be well-tolerated with no treatment-related serious adverse events. The most common adverse events, observed in less than 5% of participants, included procedural pain and itch.
The therapy is expected to be available in the third quarter of 2025, according to Abeona Therapeutics. The price is expected to be $3.1 million for the one time therapy.
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To learn more about EB and other rare skin conditions, visit https://checkrare.com/diseases/skin-conditions/