What is Fabry Disease

1. 1 What is Fabry Disease Treatment? Fabry Disease Pipeline several companies are working robustly on many new therapies, such as Lucerastat (Idorsia Pharmaceuticals), Pegunigalsidase alfa (Protalix Biotherapeutics), AVR-RD-01 (Avrobio), Venglustat (Sanofi Genzyme), ST-920 (Sangamo Therapeutics), 4D-310 (4D Molecular Therapeutics) , RVX000222 (Resverlogix Corp), and others. Mid-stage pipeline is crowded, with several potential therapies with imminent attention of big pharmaceutical companies for this Fabry Disease Market space. Lucerastat (Idorsia Pharmaceuticals) is an orally bioavailable small-molecule and low molecular weight iminosugar which inhibits glucosylceramide synthase and has the potential to provide substrate reduction therapy for Fabry disease. Idorsia Pharmaceuticals is currently conducting pivotal Phase III study for the Fabry Disease Treatment of FD. The trial would recruit up to 108 patients at sites across the U.S., Australia, Canada, and Europe with study results to be expected in June 2021.

2. 2 Besides, in phase I study, lucerastat was well-tolerated at all doses and did not cause any serious adverse events. Furthermore, Lucerastat has been granted orphan Drug designation in the US and in Europe for the treatment of Fabry disease. Pegunigalsidase alfa (Protalix Biotherapeutics) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant α-Galactosidase-A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. In clinical studies, PRX–102 has been observed to have a circulatory half-life of approximately 80 hours. In addition, Phase I/II dose-ranging studies showed high correlation between two Fabry disease biomarkers, supporting potential effectiveness of pegunigalsidase alfa in treating Fabry disease. In August 2020, the drug completed treatment period of its Phase III BRIGHT clinical trial. Additionally, the company is currently working with its service providers to complete the final monitoring visits necessary for final analysis of the data; however, many sites are still impacted by ongoing local and state restrictions and precautions due to the COVID-19 pandemic. The Company anticipates announcing top-line results in the fourth quarter of 2020 once this process is completed. AVR-RD-01 (Avrobio) it is an investigational lentiviral-based gene therapy using a harmless virus that inserts the nonmutated form of the GLA gene containing information for making functional alpha-galactosidase A into the body for the treatment of Fabery disease. It is designed to be a one-time therapy. In March 2020, company announced data from first patient treated using platoTM gene therapy platform in phase II clinical trial. According to the published results, sustained enzyme activity up to 22 months and consistent trends across multiple other measures were demonstrated. Additionally, 43 percent reduction in toxic metabolite plasma lyso-Gb3 one month post-treatment and significant higher leukocyte and plasma enzyme activity three months post-treatment than other Phase-II patients treated using academic platform at same time-point was also observed Source:- Fabry Disease Market Disease