Thanks to research, the European Medicines Agency (EMA) has recommended the approval for the first-ever topical gene therapy to help heal chronic wounds in people living with Dystrophic EB (DEB).
Vyjuvek by Krystal Biotech, Inc. is a gel that can be applied directly to wounds, targeting the root cause of the disease – the missing collagen.
Vyjuvek is a potential topical treatment that is applied directly to the wound; therefore, it is not invasive. The treatment is designed to deliver two copies of the COL7A1 gene directly to wounds. It then has the potential to treat DEB at a molecular level by helping the patient’s skin cells to make COL7 protein as normal. This therefore targets the cause of the disease.
31 patients took part in the trial with following results:
67% of patients who were treated with Vyjuvek achieved complete wound healing after 6 months, compared to only 22% who were treated with a placebo.
71% of patients who were treated with Vyjuvek achieved complete wound healing after only 3 months, compared to 20% who were treated with a placebo.
Vyjuvek was well tolerated. No drug-related serious adverse events or discontinuations due to treatment were reported. One mild drug-related adverse event was reported during the trial.
While this breakthrough brings real hope to people living with DEB worldwide, Vyjuvek is not yet available in Ireland and could take years to reach people here.
“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,” added Christine Bodemer, M.D., PhD, Professor and Head of the Department of Dermatology at the Necker Enfants Malades Hospital in Paris. “This is a remarkable advance for DEB patients and a new approach to gene therapy for genodermatoses, revolutionary and remarkably innovative.”
We will be hard at work campaigning to ensure people in Ireland get access.
That’s why we are so grateful for your support. Together, we can push for faster access to life-changing treatments for those living with EB. To keep on supporting us, please visit debra.ie/give
🧡 Thank you for standing with us!
Read more: https://lnkd.in/e-Ns9JsX and https://lnkd.in/ed5-7JAw
