Agalsidase alfa

The EMA validated an application for a less frequent Elfabrio regimen (2 mg/kg every 4 weeks) for Fabry disease, based on Phase 3 BRIGHT trial data showing its safety and effectiveness. This could reduce treatment burden for some patients.

FDA grants orphan drug designation to EXG110, a novel gene therapy for Fabry disease, aiming to improve efficacy, safety, and dosing with a 1-time treatment. The therapy targets lipid buildup in tissues, with the first patient dosed in a China-based clinical trial and plans for a US trial. Existing treatments include enzyme replacement and oral chaperone therapies, but EXG110 offers a potential simplification for patients.

Thermo Fisher Scientific expanded its CTS Detachable Dynabeads platform with new products for T-cell isolation, the FDA granted Orphan Drug Designation to Exegenesis Bio's gene therapy for Fabry disease, and Cellino Biotech appointed Chris Gemmiti as Senior Vice President of Therapeutics Development.

DelveInsight's 'Fabry Disease Pipeline Insight, 2024' report details 18+ companies developing 18+ therapies, including Venglustat, Pegunigalsidase alfa, and 4D 310, with insights on mechanism of action, route of administration, and clinical trials. Key companies include Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, and Sangamo Therapeutics. The report covers pre-clinical to marketed phases, focusing on commercial and clinical assessments.

Amicus Therapeutics and Teva Pharmaceuticals have reached an agreement allowing Teva to sell a generic version of Galafold (migalastat) for Fabry disease in the U.S. starting Jan. 30, 2037, pending FDA approval. The settlement resolves a legal dispute that began in 2020, and Amicus remains engaged in a separate dispute with Aurobindo Pharma. Galafold, a chaperone therapy, stabilizes the defective Gal A enzyme to slow Fabry disease progression, and is priced at $315,000 per year.

Elfabrio (pegunigalsidase alfa) administered every 4 weeks at 2 mg/kg appears safe and effective for stable Fabry disease adults, according to a Phase 3 study. The study, involving 30 patients, showed no severe side effects and stable kidney function, though more research is needed.

Sangamo Therapeutics announced a successful interaction with the FDA, paving the way for Accelerated Approval of its gene therapy ST-920 for Fabry disease. The FDA agreed that data from the Phase 1/2 STAAR study can support approval using eGFR slope at 52 weeks as an intermediate clinical endpoint, with a potential BLA submission anticipated in the second half of 2025.

Sangamo Therapeutics aligns with FDA for Accelerated Approval of ST-920 in Fabry disease, with BLA submission expected in 2025, using eGFR slope at 52 weeks as an intermediate clinical endpoint.