Clinical Trials

Chiesi Global Rare Diseases has announced the results from its BRIGHT trial, which evaluates the efficacy of enzyme-replacement therapy drug ELFABRIO at twice the approved dose in patients who have been treated with agalsidase alfa or beta. The current approved dose of pegunigalside alfa-iwxj (ELFABRIO) is 1 milligram (mg) per kilogram (kg) administered every two weeks.

The BRIGHT study, which was completed over the course of 52 weeks, contained 29 patients – only 21% of which were female. Results demonstrated that 30% of patients experienced at least one adverse reaction, which was mild or moderate. No patients developed novo anti-drug antibodies or treatment-emergent AEs that would have lead to a study discontinuation or mortality.

“We are pleased to share that the Journal of Inherited Metabolic Disease has published this data from this phase 3, open-label, multinational, switchover study evaluating the pharmacokinetics, safety and efficacy of 2mg/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.”

Fabry disease is a rare X-linked lysosomal disorder that results in excessive deposition of lipids in the tissues. Young patients usually present with stroke, skin lesions, heart attack, or renal failure.

“Fabry disease today is witnessing the surge of innovative research into diverse treatments, which is a new dawn of hope rising on the horizon,” said Jack Johnson, vice president, Americas & Global, Fabry International Network and co-founder, executive director Fabry Disease Support & Information Group.