Erste Gentherapie für dystrophe EB in USA zugelassen
US Food and Drug Administration (FDA) gives its first approval for new treatment for dystrophic epidermolysis bullosa
Krystal Biotech has received FDA approval for the first–ever redosable gene therapy, VYJUVEK™. The treatment is a topical gel that is applied directly onto wounds and is approved for use in patients aged six months and older. The gel treats DEB at the molecular level providing the patient’s skin cells with the template needed to restore working copies of the COL7 protein. The treatment can be administered by a healthcare professional in a clinical setting or at the patient’s home.
The approval follows a successful clinical trial that met both its primary and secondary endpoints of complete wound healing at six months and three months respectively.
The treatment aims to be available in the USA in the third quarter of 2023. Krystal aims to start the official Marketing Authorization Application procedure in Europe in the second half of 2023 with potential approval in 2024, and is currently working with the Pharmaceuticals and Medical
Devices Agency in Japan to seek approval for potential launch in 2025.
Krystal has set the wholesale price of each vial to US$24,250. The number of vials required will depend on the severity and surface area of wounds. Whilst treatment use is expected to decrease over time as the therapy decreases wound intensity and number, the initial treatment is expected to reach a steady use of 26 vials per year, equating to US$630,500.
“The FDA approval is a huge milestone for people living with DEB. This is also a gamechanger for those with rare disorders given that this is the first redosable gene therapy to have received the nod. I am very excited and remain committed to supporting initiatives for greater accessibility, and more importantly, affordability of the product for the global EB community. At DEBRA International, we will work with stakeholders to help promote access to new therapies, such as VYJUVEK™, to all patients with EB globally.” – Ritu Jain, President, DEBRA International