Neurology Advisor

Pegvaliase Gets Priority Review for PKU Treatment in Adolescents

The FDA has accepted for Priority Review the sBLA for pegvaliase-pqpz in adolescents aged 12 to 17 years with phenylketonuria.
WDAF-TV

Homology Medicines Announces Encouraging Initial Data from First Dose Level in the pheEDIT Trial Evaluating Gene Editing Candidate HMI-103 in Adults with Classical PKU

BEDFORD, Mass., July 27, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today encouraging initial clinical data from the first dose cohort in ...

FDA Accepts BioMarin's PALYNZIQ® (pegvaliase-pqpz) Supplemental Biologics License Application for Priority Review to Expand Use to Adolescents Aged 12-17 with Phenylketonuria

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the company's PALYNZIQ® (pegvaliase-pqpz) supplemental ...