Sanofi Gaucher drug approved in Europe
pharmafile | January 22, 2015 | News story | Sales and Marketing | Cerdelga, Cerezyme, Gaucher, Sanofi, eliglustat, imiglucerase
The European Commission has approved Sanofi-owned Genzyme’s Cerdelga for the treatment of Gaucher disease Type 1.
Designed for a condition that can cause spleen and liver enlargement and excessive bleeding, Cerdelga (eliglustat) has been approved for use as a first line oral therapy for a proportion of adults living with the hereditary disease.
However, a small number of patients who metabolize the drug more quickly or at an undetermined rate (as detected by a genetic lab test) will not be eligible.
The EU nod is based on two pivotal Phase III clinical trials involving around 400 patients for the drug which was approved in the US last year – and watched by analysts at Bloomberg who predict its sales could reach $749 million by 2020.
Trials showed improvements in patients’ condition including their spleen size, platelet levels, hemoglobin levels, and liver volume.
The study focussed on replacing the defective enzymes, and the results met the pre-specified criteria for non-inferiority to enzyme replacement therapy Cerezyme (imiglucerase), a drug that Sanofi has been marketing for the same disease since 2011.
Cerezyme must be infused into patients, whereas Cerdelga comes in a pill form which is usually taken twice a day, and therefore more manageable for patients.
Sanofi’s biologics unit Genzyme has priced Cerdelga at around $300,000 per year which is the same level as Cerezyme, but the French firm has not yet revealed its pricing plans for the drug in Europe.
Type 1 Gaucher is the most common form of the inherited illness, another kind being Type 2 which can cause disease or death during infancy. It affects around 10,000 people worldwide and the illness can also cause excessive bleeding and bone disease.
It can affect individuals of all ages, and defective genes – a cause of the condition – are found in one in every 100,000 people of the general population.
Tom Robinson
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