2023年05月05日，Chiesi Global Rare Diseases&Protalix BioTherapeutics, Inc.宣布,欧盟委员会 (EC) 已授予PRX-102（pegunigalsidase alfa）上市许可，用于治疗成年法布雷病患者。
欧盟委员会对PRX-102的批准是基于在超过140名患者中进行的全面临床开发计划的结果，治疗时间长达7.5年。PRX-102已在未曾接受过ERT和曾接受过ERT的患者中进行了试验，包括一个达到主要终点的头对头试验。通过评估肾小球滤过率（eGFR）的下降后发现，PRX-102在控制肾脏疾病方面显示出与获批酶替代疗法相当的疗效。
PRX-102（Pegunigalsidase alfa）聚乙二醇糖苷酶-a，是一种用于治疗法布里病的聚乙二醇化酶替代疗法，每两周注射一次，现已获欧盟批准，并正由美国FDA评估。
PRX-102是一种通过植物细胞培养表达，并经化学修饰稳定的重组α-半乳糖苷酶A。此酶蛋白质亚基通过使用短聚乙二醇（PEG）基团进行化学交联而共价结合，从而产生具有稳定药代动力学参数的分子。PRX-102在临床研究中所观察到的循环半衰期约为80小时。
法布雷病（Fabry Disease）法布里病，是一种溶酶体贮积症，由于编码α-半乳糖苷酶的基因发生突变，导致α-半乳糖苷酶A酶活性不足，而α-半乳糖苷酶A是代谢酰基鞘氨醇三己糖（Gb3）所必需的代谢酶。Gb3在细胞中的堆积会对重要器官造成严重损害，如肾脏、心脏、神经、眼睛、肠道和皮肤。目前法布里病的标准治疗为酶替代疗法（ERT）。
信息来源：https://www.prnewswire.com/news-releases/chiesi-global-rare-diseases-and-protalix-biotherapeutics-announce-european-commission-authorization-of-prx-102-pegunigalsidase-alfa-for-the-treatment-of-fabry-disease-301817012.html
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PRX-102 (Pegunigalsidase Alfa)
PRX-102 (pegunigalsidase alfa) is an experimental enzyme replacement therapy (ERT) for Fabry disease being developed by Protalix BioTherapeutics in collaboration with Chiesi Global Rare Diseases.
How PRX-102 works
People with Fabry disease are unable to make sufficient amounts of alpha-galactosidase A (Gal A), an enzyme responsible for breaking down a fatty molecule called globotriaosylceramide (Gb3). Gal A deficiency leads to the toxic accumulation of Gb3, mainly in blood vessels walls, causing damage to organs such as the kidneys, heart, and blood vessels in the brain.
Made with Protalix’s plant-based ProCellEx platform, PRX-102 delivers a modified version of Gal A directly into the bloodstream (intravenously). This version is meant to be more active and last longer in the body than others.
Replagal (agalsidase alpha) — that need to be given every two weeks. All existing ERTs are administered directly into the bloodstream.
PRX-102 in clinical trials
Two-year data from a Phase 1/2 clinical trial (NCT01678898) and its extension study (NCT01981720), involving 11 adults with Fabry disease, showed that 1 mg/kg of PRX-102, given every other week, safely and effectively lowered levels of disease biomarkers, improved kidney function, and slowed disease progression.
These promising, early findings supported the launch of three international Phase 3 clinical trials — BRIDGE (NCT03018730), BALANCE (NCT02795676), and BRIGHT (NCT03180840) — to compare PRX-102 with existing ERTs for Fabry.
The completed, switch-over BRIDGE study tested PRX-102 in 22 adults with Fabry who were previously treated with Replagal for at least two years. Participants continued on Replagal for three months of eva luations before switching to 1 mg/kg of PRX-102, given every two weeks for up to a year.
Final results showed that one year of treatment with PRX-102 was safe, and effectively slowed kidney function decline relative to that seen during Replagal’s use, meeting the trial’s main goal. This also meant that fewer patients showed moderately progressing or fast-progressing kidney disease, with 60% of patients achieving stable disease status.
PRX-102 also was found to lower the levels of lyso-Gb3 — a biomarker of Fabry disea |
