VYJUVEK, also known as B-VEC, a new treatment for dystrophic epidermolysis bullosa (DEB), is a step closer to being made available for DEB patients in the UK with the recent news that the US Food and Drug Administration (FDA) has approved it for treatment of DEB in patients aged 6 months and older in the US. Once available, VYJUVEK will be the first-ever redosable gene therapy for treatment of DEB.

VYJUVEK is a topical gel that is applied to the surface of the patient’s skin which addresses the genetic cause of DEB by restoring functional copies of the damaged genes to improve wound healing. The FDA approval was based on the results of two clinical studies which demonstrated complete wound healing at 3 and 6 months.

The European Medicines Agency (EMA) has also granted VYJUVEK orphan drug designation, a status given to certain drugs which show promise in the treatment, prevention, or diagnosis of a rare condition, such as EB. One of the benefits of this status is a simplified process for getting marketing authorisation for the EU.

Whilst this development doesn’t mean that VYJUVEK will be available for DEB patients in the UK immediately, it is a positive step towards achieving UK availability. Once the European Medicines Agency (EMA) has approved VYJUVEK for treatment of DEB in the EU it is hoped that the Medicines and Healthcare products Regulatory Agency (MHRA) will follow suit and make this treatment available for DEB patients in the UK too.

Tony Byrne – DEBRA UK CEO commented:

“On the behalf of everyone at DEBRA UK and the EB community here in the UK, I would like to pass on my congratulations to the team at Krystal Biotech on securing FDA approval for VYJUVEK. Having an approved drug treatment available for patients with DEB in the US will transform the lives of thousands of people living with this debilitating condition. We very much hope that the positive results from the clinical studies that underpinned this approval will lead to a swift approval for VYJUVEK as a treatment for DEB in the rest of the world including the UK, where DEBRA UK supports the EB community.

For more information please read the Krystal Biotech news release.