The FDA has granted a speedy review for Sanofi’s new Fabry disease pill as the next generation version of its marketed treatment Fabrazyme.
The US regulator is looking to assess GZ/SAR402671, a new investigational oral substrate reduction therapy for the treatment of Fabry disease, within the next six months.
The US regulator deemed that pill as having the potential “to treat serious or life-threatening conditions and address unmet medical needs for such diseases or conditions”.
It also provides Sanofi and its biologics arm Genzyme the opportunity to submit sections of a new drug application (NDA) on a ‘rolling basis’ before sending in a complete application.
Genzyme is currently enrolling patients in its phase II trial of GZ/SAR402671, and plans to enrol nine treatment-naïve male adult patients with Fabry disease in this study.
Fabry disease is a rare lysosomal storage disorder that results in abnormal tissue deposits of a particular fatty substance (called globotriaosylceramide, also referred to as GL-3 or Gb3) throughout the body.
GZ/SAR402671 is a glucosylceramide synthase inhibitor that blocks the formation of glucosylceramide (GL-1), a key intermediate in the synthesis of GL-3.
Through its 2011 purchase of Genzyme, Sanofi already markets a drug for the disease in the form of Fabrazyme (agalsidase beta) – a treatment that makes around $500m a year.
But even though catering for a small patient population (around 10,000 people are thought to have the disorder globally), the firm has competition in Shire’s Replagal (agalsidase alfa).
Sanofi’s new pill is the next-gen version of Fabrazyme, which is an enzyme replacement therapy and given by infusion. The French firm will hope this easier method of administration will allow it to keep its market share in the future, should it gain approval.
Genzyme’s acting head of rare diseases, 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.”
