29.06.2012 - For over a decade, Sanofi’s Cerezyme has dominated the market of therapies for Gaucher disease. Now, Shire challenges the drug with new results.
Dublin/Paris/ Carmiel/ Cambridge – Results from a head-to-head Phase III clinical study with Sanofi’s Gaucher disease therapy imiglucerase (Cerezyme) suggest that Shire’s enzyme replacement therapy velaglucerase alfa (VPRIV) offers a unique benefit to patients. According to the Irish drugmaker, only patients treated with VPRIV experienced statistically significant improvement in lumbar spine bone mineral density after nine months of treatment. This, together with a 15% lower price than Aventis‘ expensive type 1 Gaucher treatment could give the Irish an advantage in the market for the rare disease, that is narrow but lucrative.
Until VPRIV was approved in 40 countries, Aventis subsidiary Genzyme was able to charge $200,000 per year per patient for its treatment that dates back to 1997, but other players are now trying to get a piece of the cake. Pfizer and its development partner Protalix currently got US market authorisation for its plant-cell based enzyme expression therapy Elelyso (taliglucerase alfa) but it was rebuffed at the end of June by the EMA. VPRIV is made in a human cell line using Shire’s gene activation technology.
Type 1 Gaucher disease is caused by a lack of the enzyme β-glucocerebrosidase, which normally breaks down a fatty waste product called glucosylceramide. Without the enzyme, glucosylceramide builds up in the body, typically in the liver, spleen and bone marrow, which produces the symptoms of the disease: anaemia (low red blood cell counts), tiredness, easy bruising and a tendency to bleed, an enlarged spleen and liver, and bone pain and breaks. The latter symptoms are now addressed by Shires enzyme replacement therapy, according to surrogate marker data presented at the European Working Group on Gaucher Disease (EWGGD) meeting in Paris.
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