Pegvaliase

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Pegvaliase
Pegvaliase compact.png
Names
Pronunciationpeg val' i ase
Trade namesPalynziq
Other namesPegvaliase-pqpz; PEG-PAL; RAvPAL-PEG
  • (2S)-2-amino-6-[6-(2-methoxyethoxy)hexanamido]hexanoic acid
Clinical data
Drug classEnzyme[1]
Main usesPhenylketonuria[1]
Side effectsPain at injection site, joint pain, allergic reactions[1]
Pregnancy
category
Routes of
use
Subcutaneous
External links
AHFS/Drugs.comMonograph
US NLMPegvaliase
Legal
License data
Legal status
Chemical and physical data
FormulaC15H30N2O5
Molar mass318.414 g·mol−1
3D model (JSmol)
  • COCCOCCCCCC(=O)NCCCC[C@H](N)C(O)=O
  • InChI=1S/C15H30N2O5/c1-21-11-12-22-10-6-2-3-8-14(18)17-9-5-4-7-13(16)15(19)20/h13H,2-12,16H2,1H3,(H,17,18)(H,19,20)/t13-/m0/s1
  • Key:NPOCDVAOUKODSQ-ZDUSSCGKSA-N

Pegvaliase, sold under the brand name Palynziq, is a medication used to treat phenylketonuria.[1] It is used when other treatments, including diet and sapropterin, are not sufficient.[1][3] It is given by injection under the skin.[3]

Common side effects include pain at the site of injection, joint pain, and allergic reactions.[1] Other side effects may include anaphylaxis, angioedema, and serum sickness.[1][3] It is unclear if use during pregnancy is safe for the baby.[3] It is the enzyme phenylalanine ammonia lyase connected to polyethylene glycol and works by breaking down phenylalanine.[1][3]

Pegvaliase was approved for medical use in the United States in 2018 and Europe in 2019.[3][1] It is not available in the United Kingdom as of 2021.[4] In the United States it costs about 560 USD per dose up to 20 mg as of 2021.[5]

Medical uses

Dosage

It is generally started at a dose of 2.5 mg once a week for 4 weeks.[1]

This may be increased up to 60 mg once per day until phenylalanine blood levels are controlled.[1]

History

The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[6]

References

  1. 1.00 1.01 1.02 1.03 1.04 1.05 1.06 1.07 1.08 1.09 1.10 "Palynziq". Retrieved 27 October 2021.
  2. 2.0 2.1 "Palynziq". Therapeutic Goods Administration (TGA). 23 July 2021. Retrieved 5 September 2021.
  3. 3.0 3.1 3.2 3.3 3.4 3.5 "Pegvaliase-pqpz Monograph for Professionals". Drugs.com. Retrieved 27 October 2021.
  4. "Pegvaliase". SPS - Specialist Pharmacy Service. 31 March 2015. Retrieved 27 October 2021.
  5. "Palynziq Prices, Coupons & Patient Assistance Programs". Drugs.com. Retrieved 27 October 2021.
  6. New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.

External links

External sites:
Identifiers: