Feb 28 2025 Rare Disease Day 2025
Today is Rare Disease Day; a day marked around the world and an opportunity to shine a light on the 7000 rare diseases, affecting around 300 million people worldwide.
EB affects around half a million people globally. The condition is rare but the hopes and dreams of those with it are common to all of us.
In the run up to the day, we highlighted some of our Faces of EB – stories of the impact of EB on patients and their loved ones. And founder Sharmila enjoyed speaking at the Orchard Therapeutics rare disease day event.
On Rare Disease Day itself, we were delighted to receive video messages from two of our favourite professors!
Vyjuvek moves closer to approval in Europe
Good news for dystrophic EB patients as Krystal Biotech received a positive opinion for Vyjuvek for the treatment of DEB wounds from the Committee for Medicinal Products for Human Use (CHMP), an EMA committee that plays a vital role in approval of medicines in Europe.
Vyjuvek is a topical gene therapy gel and the CHMP supported application at home and in the clinic, potentially broadening its accessibility. This is an important step towards approved use of Vyjuvek to treat DEB wounds in Europe.
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. This will be the first of many launches in Europe, including a launch in France planned for later in 2025.
On Rare Disease Day, as every day, Cure EB remains dedicated to funding research to develop effective treatments, and ultimately a cure for all types of EB. Research that will fix one genetic condition will impact and inform treatment and cures for other rare genetic diseases. With your help, we will cure EB! In the words of Prof McGrath, “Let’s do more together!”