Ultragenyx Pharmaceutical has announced that US health authorities have accepted for review its resubmitted biologics licence application (BLA) for rebisufligene etisparvovec (UX111), an investigational AAV9 gene therapy for mucopolysaccharidosis type IIIA (MPS IIIA, or Sanfilippo syndrome type A). Regulators have set a target action date of 19 September 2026. If approved, the candidate would be the first therapy for this rare, progressive, and fatal paediatric neurodegenerative lysosomal storage disorder.

Rebisufligene etisparvovec is designed as a one-time intravenous infusion. It utilises a self-complementary AAV9 vector to deliver a functional copy of the sulfamidase (SGSH) gene, aiming to enable the production of the missing enzyme and reduce the accumulation of heparan sulfate. The therapy originated with Abeona Therapeutics and is now being developed by Ultragenyx. It has received multiple regulatory designations, including regenerative medicine advanced therapy (RMAT), fast track, and orphan drug (ODD) status in the US.

PharmCube's NextBiopharm® database shows that most contenders in MPS IIIA are preclinical candidates, with only four in human trials. Click here to request a free trial for NextBiopharm®.