Chiesi Global Rare Diseases has announced positive results from its BRIGHT trial, a Phase 3 study evaluating a higher dose regimen of ELFABRIO (pegunigalsidase alfa-iwxj) in patients with Fabry disease. The trial assessed the efficacy, safety, and pharmacokinetics of ELFABRIO at 2 mg/kg administered every four weeks, double the approved 1 mg/kg bi-weekly dose. The study included patients previously treated with agalsidase alfa or beta.
The BRIGHT trial, a 52-week, open-label, multinational study, enrolled 29 patients, with 21% being female. According to the company, the results, now published in the Journal of Inherited Metabolic Disease, indicate a favorable safety profile. Thirty percent of patients experienced at least one adverse reaction, which were characterized as mild or moderate. Significantly, no patients developed novo anti-drug antibodies or treatment-emergent adverse events that would have led to study discontinuation or mortality.
Fabry Disease and Current Treatment Landscape
Fabry disease is a rare X-linked lysosomal storage disorder characterized by the accumulation of lipids in tissues, leading to a range of symptoms including stroke, skin lesions, heart attack, and renal failure, often presenting in young patients. Current treatment strategies primarily involve enzyme replacement therapy (ERT) using drugs like agalsidase alfa and beta, aiming to supplement the deficient enzyme and reduce lipid accumulation.
Executive Perspectives
"We are pleased to share that the Journal of Inherited Metabolic Disease has published these data from this phase 3, open-label, multinational, switchover study evaluating the pharmacokinetics, safety and efficacy of 2 mg/kg ELFABRIO administered every four weeks," said Giacomo Chiesi, executive vice president of Chiesi Global Rare Diseases. "Chiesi is committed to evaluating additional evidence to confirm the long-term results of this administration schedule."
Jack Johnson, vice president, Americas & Global, Fabry International Network and co-founder, executive director Fabry Disease Support & Information Group, noted, "Fabry disease today is witnessing the surge of innovative research into diverse treatments, which is a new dawn of hope rising on the horizon."
