CHMP recommends approval of BioMarin's Palynziq (pegvaliase) injections for patients aged 16 and over with phenylketonuria

PKU is a rare genetic disease that manifests itself at birth and has a variety of cumulative toxic effects on the brain, characterized by the inability to break down phenylalanine (Phe), an amino acid found in various proteinsBioMarin(http:// announcedrecently that the Europeanmedicines(http://Authority (EMA) humanMedicines(http://Product
s ( http://Committee has issued an active review recommending the approval of Palynziq injections for the use of blood phenyls ( Phex) levels ( phe) despite previously available treatment optionspatients aged 16 and over with phenylketonuria (PKU) lower their blood Phe levelsin addition, CHMP noted that data collected in phase III studies and extended studies showed that patients had improved inattention and emotional symptomsCHMP's comments will now be submitted to the European Commission for review and will be decided in the second quarter of 2019about Palynziq
Palynziq, a polyethylene glycol-based recombinant phenylalanine deaminase (PAL), which was approved by DA in the United States in May 2018, becoming the first enzyme replacement therapy approved to target the underlying cause of PKU by helping the body break down Phe, the seconddrug developed and approved by BioMarin for PKU(http://CHMP's positive comments are based on the overall evaluation of data from the Palynziq Clinical Development Program, including a critical Phase III clinical study PRISM-2The study was conducted in an 8-week, double-blind, placebo-controlled, randomized drug-free study (RDT) in a group of 86 patients who were randomly accepted by Palynziq (20 mg per day or 40 mg per day) or placebo (20 mg per day or 40 mg per day)results showed that patients in the Palynziq 20mg dose group and 40 mg dose group maintained average blood Phe levels at 553.0 ?mol/L and 566.3 ?mol/L, respectively, and the baseline RT average blood Phe levels were 596.8 ?mol/L and 410.9 smol/L, respectivelyIn the placebo 20mg dose group and 40 mg dose group, the average blood Phe levels were raised to 1509.0 smol/L and 1164.0 smol/L, respectively, for the baseline RDT average blood Phe levels of 563.9?l/L and 508.2mol/L, respectivelyCHMP also considered data from the 36th month of the ongoing open label expansion study, in which patients treated with Palynziq showed a persistent maintenance of blood Phe levels, and an increase in the proportion of patients with a blood Phe threshold of normal physiological values (?120?mol/L), the recommended threshold in the United States (?360?mol/L), and an EU recommended threshold (600?mol/l)At the 36th month, 58% of patients reached phe levels of 120?mol/L, 66% of which reached less than or equal to 360?mol/L, and 72% of patients reached .600?mol/LAfter 36 months of treatment, the median protein intake from complete foods increased by 25 grams compared to baselinein addition, CHMP noted that data collected in phase III studies and extended studies showed that patients showed improved inattention and mood symptoms based on the attention deficit hyperactivity assessment scale (ADHD-RS IV) attention deficit scale (ADHD-RS IV) results assessed by the researchers and the results of the modified mood state scale (PKU-POMS) specifically for PKU