pegvaliase
Trade name: Palynziq
Recombinant proteinapprovedOrphan Drug FDAFast Track FDA
Approved
May 24, 2018
Pegvaliase, sold under the brand name Palynziq, is a medication used for the treatment of the genetic disease phenylketonuria (PKU). It is a phenylalanine (Phe)‑metabolizing enzyme. Chemically, it is a pegylated derivative of the enzyme phenylalanine ammonia-lyase that metabolizes phenylalanine to reduce its blood levels. — Wikipedia
Clinical trial activity
5 trials · 1 clinical orgs · 1 marketing orgs
Phase 1
0
Phase 2
4
Phase 3
4
Phase 4
1
Earliest trial started May 1, 2013 (NCT01819727)
Timeline
Indications
Approved for
Studied for
Mechanism of action
- PhenylalanineHYDROLYTIC ENZYME
Phenylalanine hydrolytic enzyme
Substitutes for deficient phenylalanine hydroxylase. Hydrolyses phenylalanine to reduce blood concentrations.
- Phenylalanine ammonia-lyaseSupplementation
Phenylalanine ammonia-lyase supplementation
Approval history
- approvedPriority reviewFast trackMay 24, 2018
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Parenteral
- Multi-specific
- No
Oral
No
Parenteral
Yes
Topical
No
Black box warningFirst in class
Sources
- WikipediaPegvaliase ↗
- NCATSN6UAH27EUV ↗
- ChEMBLCHEMBL4297802 ↗
Also known as
- bmn165
- bmn 165
- bmn 165
- bmn 165
- pegvaliase
- pegvaliase
- pegvaliase-pqpz
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT06780332 | Phase 4 | Jan 31, 2025 | Biomarin |
| NCT05270837 | Phase 3 | Jun 17, 2022 | Biomarin |
| NCT03694353 | Phase 3 | Sep 13, 2018 | Biomarin |
| NCT01889862 | Phase 3 | Jul 1, 2013 | Biomarin |
| NCT01819727 | Phase 3 | May 1, 2013 | Biomarin |
Showing 5 of 5 trials
Page 1 / 1
Organizations
Research & Development (1)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Biomarin | For profit | 5 | 5 | 2 | 2013 |
1 organizations
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