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Genzyme's oral Gaucher drug cleared in Europe

Cerdelga now available to adults with type 1 Gaucher disease

genzyme offices 

Patients with Gaucher disease in Europe will soon have access to a first-line, oral alternative to injectable therapy, following approval of Genzyme's Cerdelga.

Cerdelga (eliglustat) has been cleared by the European Commission (EC) for adults living with Gaucher disease type 1, according to Genzyme's parent Sanofi, which says the drug "will be available commercially in EU countries beginning in 2015 and over the next few years."

The drug is suitable for a proportion of Gaucher patients, although it should not be used to treat a group who on screening are found to be fast metabolisers of the drug, meaning they may not benefit from treatment.

Cerdelga was first approved in the US last August when it became the second oral therapy for Gaucher disease to reach the market after Actelion's Zavesca (miglustat) - although the latter drug is only approved for use after enzyme replacement therapy has failed.

Patients with Gaucher disease are currently started on injectable therapy with enzyme replacement drugs such as Genzyme's own Cerezyme (imiglucerase), the top-selling medicine for the rare disease with sales of €518m ($601m) in the first nine months of 2014.

Enzyme replacement therapy focuses on replenishing the enzyme that is deficient in Gaucher disease and breaks down the glucocerebroside that accumulates, causing symptoms. In contrast, Cerdelga and Zavesca are glucosylceramide synthase inhibitors, which work by reducing the substrate for the deficient enzyme.

In the US Genzyme has priced Cerdelga at the same level as Cerezyme - around $300,000 a year - but Sanofi has not yet revealed its pricing plans for the drug in Europe. The drug is also under regulatory review in other world markets.

Sanofi ex-chief executive Chris Viehbacher said recently that he did not expect Cerdelga to completely cannibalise Cerezyme sales, as the oral drug does not have an indication for use in children and patients already doing well on the injectable therapy will likely remain on treatment.

He added however that Cerdelga is expected to have an impact on Cerezyme and other enzyme replacement therapies for Gaucher disease, which include Shire's Vpriv (velaglucerase alfa) and Pfizer's Elelyso (taliglucerase alfa).

Analysts have suggested Cerdelga could achieve sales upwards of $350m in 2017 and eventually rival Cerezyme in peak annual sales. 

Article by
Phil Taylor

22nd January 2015

From: Sales

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