Palynziq (pegvaliase)
- Region
- 🇪🇺 EU
- Active substance
- pegvaliase
- Company
- BioMarin International Limited
- Status
- Authorised
- Therapeutic area
- Phenylketonurias
- CHMP opinion
- Thu 28 Feb 2019
- EU approval
- Fri 03 May 2019
- Designations
- Orphan
- Application
- EMEA/H/C/004744
- ATC
- A16AB19
- Official record
- www.ema.europa.eu
Indication
Palynziq is indicated for the treatment of patients with phenylketonuria (PKU) aged 16 years and older who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l) despite prior management with available treatment options.
Regulatory milestones
PALYNZIQ: PDUFA target action date (adolescent label expansion (PKU))
Biomarin Pharmaceutical Inc (BMRN); empty; status: set; "PDUFA date for the adolescent label expansion February 28, 2026; EU approval 2026 BMN 401: • Phase 3 topline data in 1 to 12 year-old population with ENPP1 deficiency 1H’26; global re"; filed 2026-02-23 · source
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Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.