In a significant development, Chiesi Global Rare Diseases has announced positive results from the Phase 3 BALANCE study, evaluating the efficacy, safety, and tolerability of pegunigalsidase alfa in adult patients with Fabry disease.
Fabry disease is a rare genetic, neurological disorder caused by a deficiency of the enzyme alpha-galactosidase A, which results in the accumulation of glycosphingolipids in various organs and tissues. This accumulation leads to a wide range of symptoms, including pain, fatigue, kidney dysfunction, and neurological and cardiovascular complications. This disease primarily affects males, but a milder and more variable form may occur in females.
The Phase 3 BALANCE study is the first randomized, double-blind, head-to-head clinical trial of enzyme replacement therapies in this patient population. The trial included 77 adults with Fabry disease who had received agalsidase beta treatment for at least one year and had deteriorating renal function. These patients were randomized in a 2:1 ratio to receive either pegunigalsidase alfa or agalsidase beta every two weeks for a duration of two years. The primary efficacy analysis assessed the annualized rate of change in estimated glomerular filtration rate (eGFR) between the two treatment arms.
The data from the Phase 3 BALANCE study demonstrated that pegunigalsidase alfa was generally well tolerated and comparable to agalsidase beta based on the rate of eGFR decline over two years. At the end of the two-year study period, the median eGFR slopes were -2.51 mL/min/1.73m²/year for pegunigalsidase alfa and -2.16 mL/min/1.73m²/year for agalsidase beta, showing comparability between the two treatment arms. Overall, pegunigalsidase alfa was generally well tolerated, with similar proportions of patients experiencing treatment-emergent adverse events and mild or moderate infusion-related reactions compared to agalsidase beta.
Fabry disease is a debilitating condition that significantly impacts the quality of life of affected individuals. The availability of pegunigalsidase alfa as a treatment option provides hope for patients. The positive results from the BALANCE study indicate that pegunigalsidase alfa can effectively slow down the decline in renal function and potentially improve tolerability compared to agalsidase beta.
For more information about Fabry disease and other rare genetic disorders, visit https://checkrare.com/diseases/congenital-and-genetic-conditions/