After nearly a decade of clinical trials, a new gene therapy called ZEVASKYN® has been approved by the Food and Drug Administration (FDA) to treat recessive dystrophic epidermolysis bullosa (RDEB) in adult and pediatric patients from birth. RDEB is a rare genetic skin disease that causes painful blistering and can have internal involvement as well.
Children’s Hospital of Philadelphia (CHOP) is the sixth Qualified Treatment Center (QTC) for the administration of ZEVASKYN® (prademagene zamikeracel) gene-modified cellular sheets, developed by Abeona Therapeutics.
Marissa J. Perman, MD, Director of the Epidermolysis Bullosa Multidisciplinary Clinic at CHOP served as a consultant to Abeona Therapeutics prior to ZEVASKYN®’s FDA approval. She expects that CHOP will administer ZEVASKYN® to three to five EB patients within the first year.
“We currently have patients in our clinic not just from the Philadelphia area, but from New York to Florida,” says Dr. Perman. “We also see patients come to us from as far away as the Middle East and South America.”
Currently no cure exists to address the underlying cause of EB. Instead, conventional treatments and gene therapies are used locally to help heal individual wounds. RDEB is the most severe subtype of EB.
The type and the severity of EB are determined by the specific gene variant that causes the disease. Different types of EB can affect different layers of skin tissue, but in general EB affects skin’s structure and strength, and the connectivity of the skin’s layers.
The common symptom of people with EB is that they have extremely fragile skin. Blisters can form in response to minor trauma, even due to rubbing the skin, and can advance to become open, bleeding sores, prone to infection and in some cases scarring. Some children also develop blisters and sores inside the body, such as in the mouth or the lining of the esophagus (food pipe). It can also affect other internal organs.
How ZEVASKYN® works
ZEVASKYN® is an ex vivo gene therapy, meaning a process in which a patient's cells from their skin biopsy are removed and modified outside of their body. ZEVASKYN® treats the recessive dystrophic form of epidermolysis bullosa (RDEB).
ZEVASKYN is a one-time therapy for targeted wounds. It can be applied on larger wound areas or on multiple smaller wounds that are on relatively flat surfaces of the body. For this therapy, a biopsy of the patient's skin is taken by a doctor, and a corrected copy of the gene is then inserted into the patient’s skin cells. Those engineered cells are then used to grow up to 12 credit-card-sized “sheets” of skin that can be grafted over the child’s open wound(s). The corrected genes in the skin graft should promote wound healing.
Patients are monitored closely and instructed to leave the treated area undisturbed for 5 to 10 days after the procedure to ensure the graft is accepted. This therapy is intended for larger wound areas as well as multiple smaller discreet wound areas on relatively flat surfaces of the body.
Dr. Perman says that in clinical trials, patients were followed for five to six years after their skin grafts were placed, and this produced “promising data on the durability of the skin grafts, meaning that the wound areas that received grafts had stayed closed.”
ZEVASKYN® builds on another approved gene therapy
This is not the first gene therapy approved for EB treatment. In May 2023, the FDA approved Vyjuvek® as a topical gene therapy treatment for patients 6 months and older with dystrophic epidermolysis bullosa (DEB). Its label has now been extended to treat all ages including newborns. Vyjuvek® delivers the corrected gene in a topically applied gel. CHOP also offers Vyjuvek® to DEB patients.
Another topical wound treatment that is not based on gene therapy, Filsuvez®, was FDA approved for children 6 months and older in December 2023.
CHOP’s leadership in gene therapy and collaborative specialty care
Patients receiving ZEVASKYN® will be treated by CHOP’s EB Multidisciplinary Clinic. What makes CHOP ideally situated to launch this new treatment? Dr. Perman cites the hospital’s longstanding global leadership in cell and gene therapies.
CHOP has one of the most experienced gene therapy teams in the world. CHOP is the only program in the United States to be a Qualified Treatment Center for all seven FDA-approved stem cell-based gene therapies for genetic blood and metabolic disorders. CHOP was directly involved in the research studies and/or clinical trials for many of them.
CHOP’s EB Multidisciplinary Clinic, open since 2017, coordinates with more than 30 different subspecialties at CHOP to address the complexities of this systemic inflammatory disease, including dermatology, sedation, surgery, gastroenterology, nursing, physical therapy, occupational therapy, psychology and more.
“This treatment requires close coordination across a number of teams, including clinical, research, administrative, financial and legal stakeholders,” Dr. Perman says. “Our clinic is already very experienced at that kind of collaborative multidisciplinary care and follow-up.”
The future of EB gene therapy
Dr. Perman hopes that ZEVASKYN® is the “next step to a more robust therapy that will, one day, lead to a cure for EB.” CHOP continues to participate in phase I and II clinical trials for other topical wound care therapies.
"We want to be the place where families go to get the specialized care they cannot get anywhere else,” Dr. Perman says.
Disclosure: Marissa J. Perman, MD, has been a consultant to Abeona Therapeutics and will be a principal investigator doing research associated with ZEVASKYN® moving forward.
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After nearly a decade of clinical trials, a new gene therapy called ZEVASKYN® has been approved by the Food and Drug Administration (FDA) to treat recessive dystrophic epidermolysis bullosa (RDEB) in adult and pediatric patients from birth. RDEB is a rare genetic skin disease that causes painful blistering and can have internal involvement as well.
Children’s Hospital of Philadelphia (CHOP) is the sixth Qualified Treatment Center (QTC) for the administration of ZEVASKYN® (prademagene zamikeracel) gene-modified cellular sheets, developed by Abeona Therapeutics.
Marissa J. Perman, MD, Director of the Epidermolysis Bullosa Multidisciplinary Clinic at CHOP served as a consultant to Abeona Therapeutics prior to ZEVASKYN®’s FDA approval. She expects that CHOP will administer ZEVASKYN® to three to five EB patients within the first year.
“We currently have patients in our clinic not just from the Philadelphia area, but from New York to Florida,” says Dr. Perman. “We also see patients come to us from as far away as the Middle East and South America.”
Currently no cure exists to address the underlying cause of EB. Instead, conventional treatments and gene therapies are used locally to help heal individual wounds. RDEB is the most severe subtype of EB.
The type and the severity of EB are determined by the specific gene variant that causes the disease. Different types of EB can affect different layers of skin tissue, but in general EB affects skin’s structure and strength, and the connectivity of the skin’s layers.
The common symptom of people with EB is that they have extremely fragile skin. Blisters can form in response to minor trauma, even due to rubbing the skin, and can advance to become open, bleeding sores, prone to infection and in some cases scarring. Some children also develop blisters and sores inside the body, such as in the mouth or the lining of the esophagus (food pipe). It can also affect other internal organs.
How ZEVASKYN® works
ZEVASKYN® is an ex vivo gene therapy, meaning a process in which a patient's cells from their skin biopsy are removed and modified outside of their body. ZEVASKYN® treats the recessive dystrophic form of epidermolysis bullosa (RDEB).
ZEVASKYN is a one-time therapy for targeted wounds. It can be applied on larger wound areas or on multiple smaller wounds that are on relatively flat surfaces of the body. For this therapy, a biopsy of the patient's skin is taken by a doctor, and a corrected copy of the gene is then inserted into the patient’s skin cells. Those engineered cells are then used to grow up to 12 credit-card-sized “sheets” of skin that can be grafted over the child’s open wound(s). The corrected genes in the skin graft should promote wound healing.
Patients are monitored closely and instructed to leave the treated area undisturbed for 5 to 10 days after the procedure to ensure the graft is accepted. This therapy is intended for larger wound areas as well as multiple smaller discreet wound areas on relatively flat surfaces of the body.
Dr. Perman says that in clinical trials, patients were followed for five to six years after their skin grafts were placed, and this produced “promising data on the durability of the skin grafts, meaning that the wound areas that received grafts had stayed closed.”
ZEVASKYN® builds on another approved gene therapy
This is not the first gene therapy approved for EB treatment. In May 2023, the FDA approved Vyjuvek® as a topical gene therapy treatment for patients 6 months and older with dystrophic epidermolysis bullosa (DEB). Its label has now been extended to treat all ages including newborns. Vyjuvek® delivers the corrected gene in a topically applied gel. CHOP also offers Vyjuvek® to DEB patients.
Another topical wound treatment that is not based on gene therapy, Filsuvez®, was FDA approved for children 6 months and older in December 2023.
CHOP’s leadership in gene therapy and collaborative specialty care
Patients receiving ZEVASKYN® will be treated by CHOP’s EB Multidisciplinary Clinic. What makes CHOP ideally situated to launch this new treatment? Dr. Perman cites the hospital’s longstanding global leadership in cell and gene therapies.
CHOP has one of the most experienced gene therapy teams in the world. CHOP is the only program in the United States to be a Qualified Treatment Center for all seven FDA-approved stem cell-based gene therapies for genetic blood and metabolic disorders. CHOP was directly involved in the research studies and/or clinical trials for many of them.
CHOP’s EB Multidisciplinary Clinic, open since 2017, coordinates with more than 30 different subspecialties at CHOP to address the complexities of this systemic inflammatory disease, including dermatology, sedation, surgery, gastroenterology, nursing, physical therapy, occupational therapy, psychology and more.
“This treatment requires close coordination across a number of teams, including clinical, research, administrative, financial and legal stakeholders,” Dr. Perman says. “Our clinic is already very experienced at that kind of collaborative multidisciplinary care and follow-up.”
The future of EB gene therapy
Dr. Perman hopes that ZEVASKYN® is the “next step to a more robust therapy that will, one day, lead to a cure for EB.” CHOP continues to participate in phase I and II clinical trials for other topical wound care therapies.
"We want to be the place where families go to get the specialized care they cannot get anywhere else,” Dr. Perman says.
Disclosure: Marissa J. Perman, MD, has been a consultant to Abeona Therapeutics and will be a principal investigator doing research associated with ZEVASKYN® moving forward.
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