Drugs / Pegvaliase
Pegvaliase
Regulatory milestones approvals, filings & regulatory actions · 7 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed phenylketonuria 3 milestones | |||||
| Filed | US (FDA) | PALYNZIQ | phenylketonuria | 2025-10-29 | “accepted for Priority Review the company's PALYNZIQ ® (pegvaliase-pqpz) supplemental Biologics License Application (sBLA) to expand treatment to include adolescents aged 12-17...” biomarin.com ↗ |
| Filed | EU (EMA) | Palynziq | phenylketonuria | 2018-03-28 | “On March 28, 2018 the European Medicines Agency accepted BioMarin's submission of a Marketing Authorization Application for pegvaliase.” biomarin.com ↗ |
| Filed | US (FDA) | — | phenylketonuria | 2017-06-30 | “announced that the company submitted a Biologics License Application (BLA) on Friday, June 30, 2017 to the U.S. Food and Drug Administration (FDA) for pegvaliase” biomarin.com ↗ “The U.S. Food and Drug Administration accepted the Biologics License Application (BLA) for pegvaliase and granted priority review status in August 2017” biomarin.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansions (2) | US (FDA) | PALYNZIQ | — | 2020-10-06 – 2026-02-27 | fda.gov ↗ |
| Approved | EU (EMA) | Palynziq | — | 2019-05-03 | europa.eu ↗ |
| Approved | US (FDA) | PALYNZIQ | — | 2018-05-24 | fda.gov ↗ |
Trials 7
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 24 trials | ||||||
| Phase 2 | NCT01560286 | May 2012 → Sep 2013 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 2 | NCT01212744 | Mar 2011 → Apr 2015 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 2 | NCT00924703 | Jan 2010 → Jan 2019 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 2 | NCT00925054 | Sep 2009 → Jun 2015 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 32 trials · 1 met primary | ||||||
| Phase 3 | NCT05270837 | Jun 2022 → Jan 2025 overdue | phenylketonuria | BioMarin Pharmaceutical | Active not recruiting | Met primary |
| Phase 3 | NCT03694353 | Sep 2018 → Jan 2021 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 41 trial | ||||||
| Phase 4 | NCT06780332 | Feb 2025 → Aug 2026 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Comparator or background therapy4 trials | ||||||
| Phase 1 | NCT00634660 Comparator | May 2008 → Apr 2009 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 3 | NCT01889862 Comparator | Jul 2013 → Jan 2016 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 3 | NCT01819727 Comparator | May 2013 → Nov 2015 | phenylketonuria | BioMarin Pharmaceutical | Completed | No outcome recorded |
| Phase 4 | NCT07477691 Background | May 2026 → Sep 2027 expected | phenylketonuria | BioMarin Pharmaceutical | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 15
| Date | Issuer | Release |
|---|---|---|
| 2026-02-27 | BioMarin Pharmaceutical | Regulatory U.S. Food and Drug Administration Approves BioMarin’s PALYNZIQ® (pegvaliase-pqpz) for Adolescents 12 Years of Age and Older with Phenylketonuria (PKU) biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has approved the company's supplemental Biologics License Application (sBLA) for PALYNZIQ ® (pegvaliase-pqpz) to include pediatric patients 12 years of age and older with phenylketonuria (PKU). |
| 2025-10-29 | BioMarin Pharmaceutical | Regulatory 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.com ↗
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 Biologics License Application (sBLA) to expand treatment to include adolescents aged 12-17 with phenylketonuria (PKU). |
| 2025-09-06 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Pivotal Phase 3 Data for PALYNZIQ® (pegvaliase-pqpz) in Adolescents with Phenylketonuria at 15th International Congress of Inborn Errors of Metabolism biomarin.com ↗
The Phase 3 PEGASUS study evaluating the efficacy and safety of PALYNZIQ in adolescents aged 12-17 demonstrated statistically significant blood phenylalanine (Phe) lowering compared to diet alone. |
| 2025-04-02 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Pivotal Data for PALYNZIQ® (pegvaliase-pqpz) in Adolescents with Phenylketonuria biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 PEGASUS trial evaluating PALYNZIQ ® (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone. |
| 2025-03-20 | BioMarin Pharmaceutical | Results BioMarin Presents New Data Demonstrating Favorable Safety and Strong Adherence in Real-World Clinical Practice with VOXZOGO® (vosoritide) in Children Under 3 with Achondroplasia at 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting biomarin.com ↗
New data also to be presented for PALYNZIQ ® (pegvaliaise-pqpz), reinforcing its value in sustaining blood Phe level reduction and improving health-related quality of life for adults with phenylketonuria |
| 2020-10-07 | BioMarin Pharmaceutical | Regulatory BioMarin, Pioneer in Rare Disease Treatments for Phenylketonuria (PKU), Receives FDA Approval of Label Expansion to Allow Maximum Dose of 60 mg for Palynziq® (pegvaliase-pqpz) Injection for Treatment of Adults with PKU biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) to increase the maximum allowable dose of 60 mg with Palynziq® (pegvaliase-pqpz) Injection for treatment of adults with Phenylketonuria (PKU). |
| 2019-05-06 | BioMarin Pharmaceutical | Regulatory European Commission Approves Palynziq® (pegvaliase injection) for Treatment of Phenylketonuria (PKU) in Patients Aged 16 Years or Older biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the European Commission (EC) has granted marketing authorization for Palynziq® (pegvaliase injection) at doses of up to 60 mg once daily, to reduce blood phenylalanine (Phe) concentrations in patients with phenylketonuria (PKU) aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options. |
| 2019-03-01 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Positive CHMP Opinion in Europe for Palynziq® (pegvaliase Injection) for Treatment of Patients with Phenylketonuria (PKU) Aged 16 and Older biomarin.com ↗ |
| 2018-05-24 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Standard Approval for Palynziq™ (pegvaliase-pqpz) Injection for Treatment of Adults with Phenylketonuria (PKU), a Rare Genetic Disease biomarin.com ↗ |
| 2018-03-28 | BioMarin Pharmaceutical | Regulatory European Medicines Agency (EMA) Accepts BioMarin’s Marketing Application for Pegvaliase MAA for Treatment of Phenylketonuria (PKU) biomarin.com ↗ |
| 2017-12-22 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Anticipated Notification of PDUFA Extension for Pegvaliase Biologics License Application (BLA) to May 28, 2018 biomarin.com ↗
In a notice received from the FDA, the Prescription Drug User Fee Act (PDUFA) Goal Date for pegvaliase has been extended by three months to May 28 , 2018. |
| 2017-09-14 | BioMarin Pharmaceutical | Regulatory FDA Not Currently Planning to Hold Advisory Committee Meeting for BioMarin’s Pegvaliase Biologics License Application (BLA) biomarin.com ↗
In the letter, the FDA advised the Company that it is not currently planning to hold an advisory committee meeting to discuss the application. |
| 2017-08-29 | BioMarin Pharmaceutical | Regulatory FDA Accepts BioMarin’s Pegvaliase Biologics License Application (BLA) and Grants Priority Review Designation biomarin.com ↗ |
| 2017-06-30 | BioMarin Pharmaceutical | Regulatory BioMarin Submits Pegvaliase Biologics License Application (BLA) to the U.S. FDA for Treatment of Phenylketonuria (PKU) biomarin.com ↗ |
| 2016-03-21 | BioMarin Pharmaceutical | Results BioMarin Phase 3 Study of Pegvaliase for Phenylketonuria (PKU) Meets Primary Endpoint of Blood Phenylalanine (Phe) Reduction (p<0.0001) biomarin.com ↗
BioMarin announced today that the pivotal Phase 3 PRISM-2 study (formerly referred to as 165-302) of pegvaliase met the primary endpoint of change in blood Phe compared with placebo (p<0.0001) in preliminary results. |
All press releases naming this drug 17 releases
Evidence & citations 10 cited values
Every value below carries the sentence it was read from. 13 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Pegvaliase | “Pegvaliase is approved to reduce phenylalanine (Phe) levels for people with phenylketonuria (PKU).” PMID 34826353 ↗ Nov 2021 |
| Known as | BMN 165 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01560286 ↗ |
| Known as | Palynziq | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06780332 ↗ |
| Known as | Peg pal | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297802 ↗ |
| Known as | Pegvaliasa | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297802 ↗ |
| Known as | Pegvaliase pqpz | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297802 ↗ |
| Known as | Phenylase | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297802 ↗ |
| Known as | rAvPAL-PEG | “BMN 165 (rAvPAL-PEG)” NCT01560286 ↗ |
| Modality | Protein / enzyme biologic | “PEGylated recombinant Anabaena variabilis phenylalanine ammonia lyase” PMID 32593547 ↗ Jun 2020 |
| Route | Subcutaneous | “subcutaneous injection” NCT00634660 ↗ |