Alice Melao,  —

Treatment with PRX-102 (pegunigalsidase alfa) can safely and effectively improve kidney function in Fabry disease patients, according to preliminary data from a Phase 3 clinical trial. One year of treatment with PRX-102 significantly improved the clinical status of all patients with progressing disease and 66.7% of those with…

Enrollment has been completed for a Phase 3 clinical trial comparing PRX-102 (pegunigalsidase alfa) with Fabrazyme (agalsidase beta) for the treatment of Fabry disease. The BALANCE trial (NCT02795676) estimated enrolling 78 Fabry disease patients with impaired renal function who had been previously treated with Sanofi Genzyme’s…

Analysis of dry blood drops may help diagnose Fabry disease and begin early treatment in patients who are experiencing cardiac, renal, or neurological symptoms of unknown cause, according to a recent study. The diagnostic and risk screening method was proposed by Japanese researchers in the study, “High-risk screening for…

Adolescents and adults with Fabry disease who live in Argentina can now be treated with Amicus Therapeutics’ oral precision medicine Galafold (migalastat). The Agency of Medicines, Food and Medical Devices (ANMAT) in Argentina has approved the use of Galafold as a monotherapy for people ages 16 or older…

Avrobio’s investigational gene therapy AVR-RD-01 shows potential in promoting durable beneficial effects to treat Fabry disease, with enhanced therapeutic activity compared with the standard-of-care enzyme replacement therapy, according to preliminary clinical data. These findings were reported after the evaluation of the first eight patients with Fabry disease who…

Protalix BioTherapeutics and Chiesi Farmaceutici are planning to submit a biologics license application with the U.S. Food and Drug Administration (FDA) for PRX-102 (pegunigalsidase alfa) as a treatment for Fabry disease, the companies have announced. After several months, and a series of meetings and correspondence with…

PRX-102 (pegunigalsidase alfa) is safe for the treatment of patients with Fabry disease and has better stability over other available therapies, according to interim data from a Phase 3 clinical trial. The most recent results from the trial were discussed during the 15th Annual WORLDSymposium 2019, recently…

Globotriaosylsphingosine (LysoGb3) blood levels can be a useful and accurate biomarker to diagnose and monitor Fabry disease, rather than only using GLA mutational status, researchers suggest. The study, “Mutations in the GLA Gene and LysoGb3: Is It Really Anderson-Fabry Disease?,” was published in the International Journal…

Inhibition of oxidative compounds using pharmacological or nutritional approaches, in addition to enzyme replacement therapy, may benefit patients with Fabry disease, research suggests. Treatment with antioxidant agents may help prevent the progressive damage in the heart and kidneys experienced by these patients. The study, “…

Chiesi, an Italian pharmaceutical company, has acquired the exclusive rights to develop and commercialize PRX-102 (pegunigalsidase alfa) in the U.S. as an investigative therapy for Fabry disease. The agreement expands upon the previously established partnership with PRX-102’s developer, Protalix BioTherapeutics, which had granted Chiesi the development and…