Sanofi's Fabrazyme: The Only ERT with Long-Term Efficacy and Safety Data for Fabry Disease

March 15, 2021 /BioValleyBIOON/ -- Sanofi Genzyme, the rare disease unit of Sanofi, recently announced a real-world observational study and a long-term treatment evaluation of Fabrazyme (agalsidase beta) in patients with Fabry disease.Clinical Trialdata, which has been included in the United StatesFDAin the approved drug labeling.

Fabrazyme received accelerated approval from the U.S. FDA in 2003, becoming the first drug approved for the treatment of adult and pediatric patients aged ≥2 years with a confirmed diagnosis of Fabry disease. Currently, Fabrazyme is the only therapy approved in the United States for the treatment of Fabry disease.FDAApproved enzyme replacement therapy (ERT) with long-term efficacy and safety data.

Dr. Robert J. Desnick, Professor and Chief of the Division of Genetics and Genomic Medicine at the Icahn School of Medicine at Mount Sinai in the United States, stated: “Fabry disease is an X-linkedHereditydisease, affecting approximately 10,000 male and female patients worldwide. These findings make a significant contribution to our ongoing understanding of this rare disease and its long-term impact on renal function and major clinical events.”

In a randomized (Fabrazyme: placebo = 2:1), double-blind, placebo-controlled, multinational, multicenter clinical study, 82 adult patients with Fabry disease who had not previously received enzyme replacement therapy were evaluated for the time to first occurrence of a clinically significant event (renal, cardiac, or cerebrovascular events, or death). Patients received treatment with Fabrazyme or placebo for up to 35 months (median follow-up: 18.5 months). Clinically significant events occurred in 14 out of 51 patients (28%) receiving Fabrazyme and in 13 out of 31 patients (42%) receiving placebo. Compared with the placebo group, the risk of experiencing a clinically significant event was reduced by 43% in the Fabrazyme group (HR = 0.57; 95% CI: 0.27–1.22).

A long-term observational study evaluated the rate of decline in renal function (eGFR) in 122 patients (aged 16 years or older) treated with Fabrazyme, and compared them with a historical cohort of 122 untreated patients matched 1:1 based on age, sex, classic or non-classic Fabry disease subtype, and baseline estimated eGFR. The median follow-up time was 3 years for the untreated group and 4.5 years for the treated group (with a maximum of 5 years for both groups). In the Fabrazyme-treated group, the estimated mean slope of eGFR was -1.5 mL/min/1.73 m²/year, compared to -3.2 mL/min/1.73 m²/year in the untreated group, resulting in a treatment difference of 1.7 mL/min/1.73 m²/year (95% CI: 0.5–3.0).

Alaa Hamed, Global Head of Medical Affairs for Rare Diseases at Sanofi, stated, “We extend our deepest gratitude to the thousands of patients, physicians, and researchers who have contributed to advancing our understanding of Fabry disease over the past 20 years. We believe this evidence is built upon a comprehensive understanding of the efficacy and safety profile of Fabrazyme.”

Fabry disease is an X-linkedHeredityX-linked disorder with the defective gene located on the long arm of the X chromosome. This disease is caused by a defect in the α-galactosidase A (GLA) gene, resulting in deficient activity of lysosomal α-galactosidase, which leads to the progressive accumulation of a lipid substance called globotriaosylceramide (Gb3) in the vascular walls throughout the body.

Fabry disease is a rare disorder, affecting one in every 40,000 to 60,000 individuals. Patients have a deficiency in alpha-galactosidase A, the enzyme responsible for the breakdown of globotriaosylceramide (Gb3). The abnormal accumulation of Gb3 increases over time, leading to its predominant deposition in the blood and vascular walls. The ultimate consequences of Gb3 deposition range from pain and impaired peripheral sensation to organ failure, particularly renal failure, as well as cardiac and cerebrovascular complications.

Fabrazyme works by replacing the naturally occurring enzyme (alpha-galactosidase A) to help clear accumulated Gb3 from cells, including those in the blood vessels of the kidneys, heart, and skin. Fabrazyme can be used for treatment regardless of genotype, disease severity, or enzyme activity levels. In patients receiving Fabrazyme treatment, the most common adverse reactions with an incidence rate more than 2.5% higher than that of placebo areAdverse ReactionsUpper respiratory tract infection, chills, fever, headache, cough, burning or tingling sensations in the hands/arms/legs or feet, fatigue, lower extremity edema due to fluid accumulation, dizziness, and rash. (Bioon.com)

Original Source: Long-term data on Fabrazyme® (agalsidase beta) for people with Fabry disease