Commercial-stage biopharmaceutical company developing small-molecule therapies for rare and neurodegenerative diseases; its lead drug AQNEURSA (levacetylleucine) is FDA-approved for Niemann-Pick disease type C and, as of September 2026, ataxia-telangiectasia — the first approved treatment for that disease anywhere.
Commercial rollout of AQNEURSA for ataxia-telangiectasia in the U.S.
IntraBio has secured FDA approval to expand the label of AQNEURSA (levacetylleucine), making it the first treatment in the world indicated for ataxia-telangiectasia and a second approved indication for the chemically altered amino acid after Niemann-Pick disease type C.
Founder and Executive Chairman