Sanofi Fabrazyme clinical trial progress announced

Sanofi Genzyme, a rare disease unit of Sanofi, recently announced a real-world observational study and a long-term observational study evaluating Fabrazyme (agalsidase beta) in the treatment of Fabry disease patients.

Fabrazyme received accelerated approval from the US FDA in 2003 and became the first drug for the treatment of adults and children aged ≥ 2 years who were diagnosed with Fabry disease.

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In a randomized (Fabrazyme: placebo=2:1), double-blind, placebo-controlled, multi-country, multi-center clinical study, 82 adult patients with Fabry disease who did not receive enzyme replacement therapy were evaluated.

A long-term observational study evaluated the renal function decline rate (eGFR) of 122 patients (age: 16 years or older) treated with Fabrazyme, and compared it with a historical cohort of 122 untreated patients (based on age, gender, typical or SARS).

Fabry disease (Fabry disease) is an X-linked genetic disease in which the defective gene is located on the long arm of the X chromosome.

Fabry disease is a rare disease, affecting one person in every 40,000-60,000.

The working principle of Fabrazyme is to replace the naturally occurring enzyme (α-galactosidase A) to help clear the accumulation of Gb3 in cells, including blood vessels in the kidneys, heart and skin.

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

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