NEW YORK--(BUSINESS WIRE)--400 million people around the world are affected by a rare disease. 95% of rare diseases lack an FDA approved treatment or cure. Today, DEB warriors around the world cross into the 5% with the approval of Krystal Biotech’s VYJUVEK™ treatment. VYJUVEK is a non-invasive, topical, redosable gene therapy.
A HISTORIC APPROVAL FOR THE EB COMMUNITY
EB Research Partnership (EBRP) is the largest global funder of innovative Epidermolysis Bullosa (EB) research and development with a mission to rapidly accelerate treatments and achieve a cure by 2030. Today, people with EB around the world receive a glimpse into that future.
"My family has been dreaming of this day since our daughter Elodie was born almost 7 years ago. This is a remarkable milestone for families across the world, who now have a tool to treat their loved ones' pain. We believe this approval will also help pave the way for a cure, which remains elusive. We want to express our unending gratitude to the families, doctors and researchers who worked tirelessly to make this day possible, before we had even heard of EB,” says Emily Kubik, a parent to 6-year-old Elodie Kubik who was born with Recessive Dystrophic Epidermolysis Bullosa (RDEB) and an EBRP Board Member.
This is not only the first-ever FDA approved therapy for those battling DEB in the US, but also the first-ever FDA approved topical, redosable gene therapy (Source).
A SCALABLE MODEL THAT’S BREAKING GROUND
This FDA approval marks a milestone in EBRP’s mission to discover treatments and a cure for EB. It provides validation for their scalable and sustainable Venture Philanthropy business model and most importantly creates hope for families around the world battling EB every day.
EBRP’s Venture Philanthropy business model has been highlighted for its leadership by Harvard Business School, Yale University, and MIT in helping to fast-track not only a cure for EB but therapies that could affect thousands of other rare diseases.
EBRP invested in Krystal Biotech in 2017, shortly after Krystal Biotech’s initial public offering of common stock. The investment was made via a private placement of Krystal Biotech’s common stock, and as part of the stock purchase agreement, Krystal Biotech committed to commence a Phase I clinical trial of VYJUVEK. The funding followed the highly competitive application and screening process overseen by EBRP’s Scientific Advisory Board (SAB), which is composed of leading scientists and physicians. EBRP was able to generate a more than double return on its investment and reinvest that capital back into more EB projects.
"The FDA approval of Krystal Biotech’s VYJUVEK marks a historic day for the EB community. We are grateful for Krystal Biotech’s commitment to delivering the first ever FDA approved treatment for DEB. Our investment in Krystal Biotech provides a strong case for our leading Venture Philanthropy model in action. Having worked in service to the patient community for 20 years, these are the wins that we relentlessly fight for everyday. And tomorrow, we will get up and continue fighting until there is a treatment for every patient and we achieve our ultimate goal of a cure,” says Michael Hund, CEO of EBRP.
A COMMUNITY COMMITTED TO FINDING A CURE
EB is a group of devastating and life-threatening skin disorders that affects 500,000 people worldwide (Source). DEB is a rare and severe form of EB. Called "Butterfly Children" because their skin is as fragile as a butterfly's wings, children with DEB face severe pain, open external and internal wounds, and a grueling daily bandaging process.
Since its inception EBRP has:
- Raised over $50M
- Funded 120+ EB projects
- Contributed to a 20x growth in the EB clinical trial landscape
Founded by a group of dedicated parents along with Jill and Eddie Vedder (Pearl Jam), EBRP is the largest nonprofit dedicated to funding research aimed at treating and ultimately curing EB. Today’s FDA approval of the first-ever medication to treat DEB is an exciting milestone on this journey. The organization is committed to building off this momentum and delivering a cure.
For more information on how to donate and get involved can be found at ebresearch.org. You can follow @ebresesarch on Instagram, Facebook, LinkedIn and Twitter.
About EB Research Partnership
Founded in 2010 by a dedicated group of parents and Jill and Eddie Vedder, EB Research Partnership (EBRP) is the largest global nonprofit dedicated to funding research aimed at treating and ultimately curing Epidermolysis Bullosa (EB), a group of devastating and life-threatening skin disorders that affect children from birth.
Working around the clock with offices in the US and Australia, EBRP utilizes an innovative Venture Philanthropy business model. When making a grant to a research project, they retain the added upside of generating a recurring revenue stream if the therapy or product is commercially successful, then use the return on investment to fund additional EB research until a cure is found.
To learn more, visit www.ebresearch.org
