CHMP recommends Vyjuvek OK for DEB patients from birth
Therapy gives healthy gene copies to cells with instructions to produce collagen
A committee of the European Medicines Agency is recommending approving Vyjuvek (beremagene geperpavec), a gene therapy from Krystal Biotech, to treat wounds in patients of all ages with dystrophic epidermolysis bullosa (DEB).
The opinion by the Committee for Medicinal Products for Human Use (CHMP) will now go to the European Commission for a final decision on marketing authorization. That’s expected in the second quarter of this year and will apply to all European Union countries, plus Iceland, Norway, and Liechtenstein.
“The CHMP’s recommendation for approval of Vyjuvek is an exciting step toward our goal of delivering the first ever [gene] therapy to DEB patients across Europe,” Suma Krishnan, Krystal’s president of research and development, said in a company press release.
Vyjuvek was approved in the U.S. in 2023 as the first gene therapy to apply topically on wounds of DEB patients 6 months and older. It’s available in the U.S. for use by healthcare professionals in a clinic or at home.
“Our team in Europe has been working tirelessly in preparation for Krystal’s first commercial launch outside of the United States and with the CHMP’s positive opinion we remain on track to launch in Germany around the middle of this year,” said Laurent Goux, Krystal’s senior vice president and general manager of Europe.
If the European Commission decides on marketing authorization, “this will be the first of many launches in Europe, including a launch in France planned for later in 2025, as we advance diligently to ensure as many patients as possible benefit from access to Vyjuvek,” Goux said.
How Vyjuvek works on DEB
Vyjuvek comes as a suspension that must be mixed into a gel before it’s used. Dosing can be adjusted depending on the patient’s age and how large their wounds have grown.
“The CHMP’s support for a broad label, including treatment of patients from birth and the option of patient or caregiver administration at home, are also fantastic outcomes for the DEB patients we aim to serve, broadening access and reducing barriers to starting on and staying on therapy,” Krishnan said.
Epidermolysis bullosa makes the skin become fragile and likely to blister and wound in response to even minimal friction. In DEB, this is caused by mutations in COL7A1, a gene that contains instructions for producing part of a protein called type VII collagen, which is essential for holding layers of skin onto each other.
Applied in droplets on wounds once weekly, Vyjuvek delivers healthy copies of COL7A1 directly into skin cells, providing them with instructions to produce type VII collagen, which should help heal the wounds.
“By addressing the very first stage in the complex [disease mechanism] of DEB, Vyjuvek is a landmark. It is amazing how simple and noninvasive its use is, even in infants,” said Cristina Has, MD, a professor and dermatologist at the University of Freiburg in Germany. This could make “a true difference in their lives,” Has said.
The CHMP’s recommendation drew on positive data from the Phase 1/2 GEM-1 study (NCT03536143) and the Phase 3 GEM-3 study (NCT04491604), where Vyjuvek outperformed a placebo at bringing about complete wound healing after three and six months. The therapy’s long-term safety and efficacy are also supported by data from a completed open-label extension study (NCT04917874), where patients were followed for up to two years, along with real-world experiences with Vyjuvek since its U.S. launch.
“We are very pleased that our patients, from birth, will have a simple, topical treatment that promotes durable wound closure, something that until now has been beyond the reach of any therapy,” said Christine Bodemer, MD, PhD, who heads the dermatology department at the Necker Enfants Malades Hospital in Paris. “This is a remarkable advance for DEB patients.”
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