Pegvaliase

Pegvaliase

(2S)-2-amino-6-[6-(2-methoxyethoxy)hexanoylamino]hexanoic acid

CAS 1585984-95-7

• Molecular FormulaC₁₅H₃₀N₂O₅
• Average mass318.409 Da

BMN-165

Palynziq

pegvaliase

pegvaliase-pqpz

L-Lysine, N⁶-[6-(2-methoxyethoxy)-1-oxohexyl]-

N⁶-[6-(2-Methoxyethoxy)hexanoyl]-L-lysine 

AUSTRALIA APPROVAL 2021

PALYNZIQ

Evaluation commenced: 30 Sep 2020

Registration decision: 6 Jul 2021

Date registered: 14 Jul 2021

Approval time: 166 (175 working days)

pegvaliase

BioMarin Pharmaceutical Australia Pty Ltd

PALYNZIQ (solution for injection, pre-filled syringe) is indicated for the treatment of patients with phenylketonuria (PKU) aged 16 years and older who have inadequate blood phenylalanine control despite prior management with available treatment options.

Pegvaliase, sold under the brand name Palynziq, is a medication for the treatment of the genetic disease phenylketonuria.^[2][3] Chemically, it is a pegylated derivative of the enzyme phenylalanine ammonia-lyase that metabolizes phenylalanine to reduce its blood levels.^[4]

It was approved by the Food and Drug Administration for use in the United States in 2018.^[2] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.^[5]

Pegvaliase is a recombinant phenylalanine ammonia lyase (PAL) enzyme derived from Anabaena variabilis that converts phenylalanine to ammonia and trans-cinnamic acid. Both the U.S. Food and Drug Administration and European Medicines Agency approved pegvaliase-pqpz in May 2018 for the treatment of adult patients with phenylketonuria (PKU). Phenylketonuria is a rare autosomal recessive disorder that is characterized by deficiency of the enzyme phenylalanine hydroxylase (PAH) and affects about 1 in 10,000 to 15,000 people in the United States. PAH deficiency and inability to break down an amino acid phenylalanine (Phe) leads to elevated blood phenylalanine concentrations and accumulation of neurotoxic Phe in the brain, causing chronic intellectual, neurodevelopmental and psychiatric disabilities if untreated. Individuals with PKU also need to be under a strictly restricted diet as Phe is present in foods and products with high-intensity sweeteners. The primary goal of lifelong treatment of PKU, as recommended by the American College of Medical Genetics and Genomics (ACMG) guidelines, is to maintain blood Phe concentration in the range of 120 µmol/L to 3690 µmol/L. Pegvaliase-pqpz, or PEGylated pegvaliase, is used as a novel enzyme substitution therapy and is marketed as Palynziq for subcutanoues injection. It is advantageous over currently available management therapies for PKU, such as [DB00360], that are ineffective to many patients due to long-term adherence issues or inadequate Phe-lowering effects. The presence of a PEG moiety in pegvaliase-pqpz allows a reduced immune response and improved pharmacodynamic stability.

References

1. ^ Jump up to:^a b “Palynziq”. Therapeutic Goods Administration (TGA). 23 July 2021. Retrieved 5 September 2021.
2. ^ Jump up to:^a b “FDA approves a new treatment for PKU, a rare and serious genetic disease” (Press release). Food and Drug Administration. May 24, 2018.
3. ^ Mahan KC, Gandhi MA, Anand S (April 2019). “Pegvaliase: a novel treatment option for adults with phenylketonuria”. Current Medical Research and Opinion. 35 (4): 647–651. doi:10.1080/03007995.2018.1528215. PMID 30247930.
4. ^ “Palynziq”. BioMarin Pharmaceutica.
5. ^ New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.

External links

• “Pegvaliase”. Drug Information Portal. U.S. National Library of Medicine.

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