Sanofi and Genzyme’s new Gaucher disease drug recommended by CHMP

Sanofi and Genzyme have announced that their new Gaucher disease treatment Cerdelga has been recommended for European regulatory approval.

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion on the approval of the oral treatment for certain adults living with type 1 Gaucher disease, with a final decision expected in the coming months.

Data from the Cerdelga clinical development programme has demonstrated the safety and efficacy of the drug. It was the largest programme of studies ever conducted for Gaucher disease, with approximately 400 patients treated in 29 countries.

Genzyme has been researching an oral therapy for Gaucher disease for around 15 years. Cerdelga has already been approved by US regulators, with application processes ongoing in a number of other countries.

Dr David Meeker, Genzyme's president and chief executive officer, said: "With the availability of an effective oral therapy, Cerdelga would offer some patients and their physicians more choice in how to manage this serious disease."

Genzyme has been part of Sanofi since 2011. Together, they have developed a number of treatments for multiple sclerosis – including Lemtrada and Aubagio – in addition to their work on Cerdelga.