Sanofi group Genzyme will be celebrating news that US regulators have awarded fast-track designation to its investigational treatment for the rare genetic disorder Fabry disease, which affects around 10,000 people around the globe.
The rare lysosomal storage disorder is characterised by excessive accumulation of a fatty substance - glucosylceramide, also known as GL-3 or Gb3 - throughout the body, potentially leading to life threatening renal, cardiac and cerebrovascular events.
Genzyme’s GZ/SAR402671 is a glucosylceramide synthase inhibitor that works by blocking the formation of glucosylceramide (GL-1), a key component in the production of GL-3, currently being tested in Phase II trials.
The US Food and Drug Administration’s fast-track programme is designed to facilitate frequent interactions between the company and review team to accelerate the clinical development and availability of new medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs.
Genzyme already markets the enzyme replacement therapy Fabrazyme (agalsidase beta) in the US, which became the first treatment approved for the disease back in 2003.