Pegvaliase

Pegvaliase
Names
Pronunciationpeg val' i ase
Trade namesPalynziq
Other namesPegvaliase-pqpz; PEG-PAL; RAvPAL-PEG
IUPAC name
  • (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
    • AU: S4 (Prescription only) [2]
    • US: ℞-only
    • EU: Rx-only
    Chemical and physical data
    FormulaC15H30N2O5
    Molar mass318.414 g·mol−1
    3D model (JSmol)
    SMILES
    • COCCOCCCCCC(=O)NCCCC[C@H](N)C(O)=O
    InChI
    • 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 2 3 4 5 6 7 8 9 10 11 "Palynziq". Archived from the original on 20 October 2021. Retrieved 27 October 2021.
    2. 1 2 "Palynziq". Therapeutic Goods Administration (TGA). 23 July 2021. Archived from the original on 5 September 2021. Retrieved 5 September 2021.
    3. 1 2 3 4 5 6 "Pegvaliase-pqpz Monograph for Professionals". Drugs.com. Retrieved 27 October 2021.
    4. ↑ "Pegvaliase". SPS - Specialist Pharmacy Service. 31 March 2015. Archived from the original on 27 October 2021. Retrieved 27 October 2021.
    5. ↑ "Palynziq Prices, Coupons & Patient Assistance Programs". Drugs.com. Archived from the original on 26 January 2021. Retrieved 27 October 2021.
    6. ↑ New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Archived from the original on 17 September 2020. Retrieved 16 September 2020.
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