Genzyme

Sanofi company Genzyme has received fast-track designation from the US Food and Drug Administration (FDA) to develop its GZ/SAR402671, a new investigational oral substrate reduction therapy to treat Fabry disease.

Fabry disease is a rare lysosomal storage disorder, which results in abnormal tissue deposits of a particular fatty substance called globotriaosylceramide (GL-3 or Gb3) throughout the body.

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It can be characterised by excessive accumulation of the lipid GL-3 in various organs and tissues and it may result in life-threatening renal, cardiac and cerebrovascular events.

GZ/SAR402671 is a glucosylceramide synthase inhibitor, which blocks the formation of glucosylceramide (GL-1), a key intermediate in the synthesis of GL-3.

“GZ/SAR402671 is a glucosylceramide synthase inhibitor, which blocks the formation of glucosylceramide (GL-1), a key intermediate in the synthesis of GL-3.”

Genzyme rare diseases acting head Dr Richard Peters said: “Becoming a fast-track programme is an important milestone and we appreciate this designation from FDA.

“We look forward to learning more about this small molecule, with the goal of providing more therapeutic options to the Fabry community as quickly as possible.”

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Currently, the company is enrolling patients in its Phase IIa trial for GZ/SAR402671 and is planning to enrol nine treatment-naïve male adult patients with Fabry disease in the international multicenter study.

In February this year, Genzyme partnered with Voyager Therapeutics to discover, develop and commercialise adeno-associated virus (AAV) gene therapies for severe central nervous system (CNS) disorders.

The alliance will carry out multiple gene therapy programmes, including programmes for Parkinson’s, Friedreich’s ataxia and Huntington’s disease, as well as other CNS disorders.

Image: The entrance to the Genzyme building at 500 Kendall Square in Cambridge, Massachusetts. Photo: courtesy of Tim Pierce.