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FDA gives green light to Genzyme’s oral Gaucher drug

Oral therapy Cerdelga is set to complement intravenous Cerezyme
FDA headquarters White Oak

Sanofi's Genzyme subsidiary has won approval in the US for Cerdelga, which it says is the only first-line oral therapy for adults with type 1 Gaucher disease.

Genzyme has said it plans to launch Cerdelga (eliglustat), a drug in the glucosylceramide class, within the next month, giving it an oral therapy to suit alongside its widely-used enzyme replacement therapy Cerezyme (imiglucerase) which is administered by intravenous infusion.

It is not the first oral drug for Gaucher on the market - that distinction goes to Actelion's Zavesca (miglustat) - although this therapy is only approved for use after enzyme replacement therapy has failed.

Cerdelga, which was granted a priority review by the FDA, is “another important treatment option” for Gaucher disease, according to the agency's deputy director of the Office for Drug Evaluation II, Amy Egan.

Patients with Gaucher disease accumulate lipid engorged cells around the body, predominantly in the spleen, liver and bone marrow, which can lead to spleen and liver enlargement, anaemia, excessive bleeding and bruising and bone disease.

There are around 6,000 people in the US eligible to receive treatment with Cerdelga, which was approved on the back of two phase III trials involving a total of 199 patients.

The ENGAGE trial – in 40 patients who had not been treated before with enzyme replacement therapy - showed that a twice-daily dose of the drug reduced spleen volume significantly compared to placebo after 39 weeks, and also improved liver volumes, blood platelet counts and haemoglobin levels.

The second study, called ENCORE, compared Cerdelga to Cerezyme in 159 patients who had been on enzyme replacement for at least three years and found that the oral therapy performed as well as the comparator drug, which is given as an intravenous infusion.

Cerezyme is already the biggest-selling drug for Gaucher disease worldwide, bringing in €688m (around $913m) in net sales last year, and eliglustat is expected to be an important addition to the company's franchise, which competes with rival drugs such as Shire's Vpriv and Pfizer's Elelyso (taliglucerase alfa).

The drug acts at a level above the defective enzyme in Gaucher disease and reduces the amount of a toxic metabolite (glucosylceramide) that is the target of enzyme replacement therapy, raising the possibility that Cerdelga could be used alongside Cerezyme as a combination regimen.

Genzyme chief executive David Meeker said the company intends to price Cerdelga at around the same level as Cerezyme, which at $300,000 per year is one of the priciest drugs on the market.

The company said it wants a pricing strategy that does not drive patients towards either Cerezyme or Cerdelga, making the choice of therapy a purely clinical decision.

Article by
Phil Taylor

21st August 2014

From: Sales

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