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news.Abeona Therapeutics has announced receipt of the US Food and Drug Administration (FDA) approval for gene-modified cellular sheet therapy Zevaskyn for the wound treatment in both adult and paediatric patients suffering from recessive dystrophic epidermolysis bullosa (RDEB), a serious genetic skin condition.
In the trial, the therapy showed 50% or more healing from baseline in large chronic RDEB wounds. Credit: saulhm on Pixabay.
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According to the company, Zevaskyn is the first and only autologous cell-based gene therapy.
The FDA’s decision is supported by the outcomes from the pivotal randomised intrapatient-controlled multi-centre Phase III VIITAL trial.
In the trial, Zevaskyn is claimed to have shown 50% or more healing from baseline in large chronic RDEB wounds, alongside a decrease in pain, as measured by the Wong-Baker FACES scale at six months post-treatment.
Abeona noted that Zevaskyn is anticipated to become available at the start of the third quarter of this year via a network of Zevaskyn Qualified Treatment Centers (QTCs).
The therapy is said to comprise the patient’s skin cells, known as keratinocytes, which have undergone genetic modification to create functional Type VII collagen.
These sheets of Zevaskyn are then surgically placed onto the wounded region of the patients.
A single application can involve the use of up to 12 sheets, each roughly the size of a credit card, which can be combined to cover extensive regions or used on various distinct wounds, providing substantial coverage for the damaged regions of the body.
An open-label single-centre Phase I/IIa trial, involving 38 chronic wounds across seven subjects, indicated that the therapy’s one surgical application could lead to improvement on a long-term basis at treated areas, with a median follow-up period of 6.9 years.
Abeona Therapeutics CEO Vish Seshadri said: “Today’s approval of Zevaskyn represents a pivotal moment in the treatment of RDEB, answering the call of people living with the clinical, economic, and human impact of this devastating disease.
“Through a single surgical application, Zevaskyn can now offer people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds, as evidenced by the results from our pivotal Phase III study.”
In conjunction with the agency’s approval, the company has been awarded a rare paediatric disease priority review voucher (PRV) and announced its intention to monetise this voucher.