• Estimated Number of patients in the United States: Approximately 25,000 ~ 50,000 patients. 40% of the patients with EB have dystrophic or junctional type of EB
Most die before 10 years old.
• A rare disease that requires high medical care cost for treatment
The cost for treatments of symptoms that appear on the skin exceed $30,000/year.
Expenses for products of treating injury that is not supported by insurance and governmental program exceeds $5,000/month per patient.
Reference: Annual payment for orphan drug per patient is hundreds of thousands dollars
• Ivacaftor (Kalydeco), a treatment of cystic fibrosis: $325,000
• Eculizumab (Soliris), a treatment of paroxysmal nocturnal hemoglobinuria: $486,000