BridgeBio Pharma Receives FDA Fast Track Designation for Investigational Gene Therapy for Congenital Adrenal Hyperplasia
The FDA\xe2\x80\x99s Fast Track designation reinforces the urgency to address the unmet needs of patients with CAH as quickly and safely as possible.
- The FDA\xe2\x80\x99s Fast Track designation reinforces the urgency to address the unmet needs of patients with CAH as quickly and safely as possible.
- We are grateful to have received Fast Track along with other key designations granted by the FDA and the EMA,\xe2\x80\x9d said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy, which is focused on developing gene therapy treatment options for patients in need.
- The disease is caused by deleterious mutations in the gene encoding an enzyme called 21-hydroxylase, leading to lack of endogenous cortisol and aldosterone production.
- The presentation covered three key Investigational New Drug (IND)-enabling studies that informed the anticipated upcoming clinical trial for BBP-631.