Palynziq injections obtain FDA standard approval for PKU adult patients
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Last Update: 2020-06-11
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Source: Internet
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Author: User
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PKU is a rare genetic disease that manifests itself at birth and can lead to various cumulative toxicity to the brainrecently, BioMarin Pharmaceutical announced that the Palynziq (pegvaliase-pqpz) injection has been approved by the U.SFDA(standard approval) to reduce blood phenylalanine (Phe) levels in adult patients with phenylketonuria (PKU)this is the first enzyme replacement therapy to be approved to target the underlying causes of PKU by helping the body break down PheIt is also BioMarin's second approved treatment for this important conditionabout Palynziq
Palynziq is a polyethylene glycol-based recombinant phenylalanine deaminase that is used to replace the phenylalanine hydroxylase (PAH) deficiency in PKU patients to break down PheIts effects are(in clinical trials)In the key 3 study PRISM-2, Palynziq significantly reduced blood Phe levels (p0.0001) compared to placebo to reach the primary endpoint of blood Phe changesin PRISM-2 double-blind, placebo-controlled, randomized discontinuation trials (RWP), patients were assigned a 2:1 ratio to continue to receive Palynziq therapy (20 mg per day or 40 mg per day) or continued to receive a placebo for 8 weeksresults showed that patients in the Palynziq group were able to maintain blood Phe concentrations, while blood Phe concentrations in placebo group patients were restored to pre-treatment baselines
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