First enzyme replacement therapy approved by European Union for phenylketonuria
-
Last Update: 2019-05-07
-
Source: Internet
-
Author: User
Search more information of high quality chemicals, good prices and reliable suppliers, visit
www.echemi.com
Today, biomarin pharmaceutical announced that the European Union approved the launch of its palynziq (pegvaliase injection) to reduce the blood phenylalanine (PHE) level in patients over 16 years old with phenylketonuria (PKU) The levels of Phe in the blood of these patients were not sufficiently controlled even with other treatments Palynziq is a PEGylated phenylalanine ammonia lyase (PAL), which is the first approved enzyme replacement therapy targeting PKU pathological mechanism in Europe PKU is a rare genetic disease Due to the inability of patients to degrade Phe, this amino acid accumulates in the body and produces toxicity to the brain If not treated, the toxicity of Phe to the brain can lead to serious neurological and neuropsychological problems Because of the severity of the disease's symptoms, babies in many countries are screened at birth to ensure early diagnosis and treatment of PKU PKU patients need special diet and other methods to control their Phe level for life Palynziq is a PEGylated phenylalanine ammonia lyase, which can degrade Phe to reduce the level of Phe in patients' blood to the target range EU approval for palynziq is based on the results of its clinical development project, which includes a phase 3 clinical trial called prism-2 The results of this trial showed that patients treated with 20 or 40 mg of palynziq were able to maintain blood Phe levels at 553.0 μ mol / L and 566.3 μ mol / L after 8 weeks The levels of the 20 mg or 40 mg placebo group were 1509.0 μ mol / L and 1164.0 μ mol / L In the 36 month open label extension study, patients using palynziq had a sustained decrease in blood Phe levels The mean Phe level decreased from 1233 μ mol / L at baseline to 565 μ mol / L (month 12, n = 164), 345 μ mol / L (month 24, n = 90), and 341 μ mol / L (month 36, n = 48) After 36 months, 72% of the patients reached the treatment target recommended by the European Union (PHE level lower than 600 μ mol / L) "The approval of palynziq is the latest milestone in our commitment to PKU patients for 15 years Biomarin brings only two approved treatments to PKU patients around the world We will continue to use our expertise in PKU to further promote standard therapies for this serious and rare disease " Mr Jean Jacques bienaim é, chairman and chief executive officer of biomarin, said
This article is an English version of an article which is originally in the Chinese language on echemi.com and is provided for information purposes only.
This website makes no representation or warranty of any kind, either expressed or implied, as to the accuracy, completeness ownership or reliability of
the article or any translations thereof. If you have any concerns or complaints relating to the article, please send an email, providing a detailed
description of the concern or complaint, to
service@echemi.com. A staff member will contact you within 5 working days. Once verified, infringing content
will be removed immediately.