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FDA approves Sanofi’s new rare disease drug

pharmafile | August 20, 2014 | News story | Research and Development, Sales and Marketing Elelyso, Gaucher disease, Genzyme, Sanofi, eliglustat 

Sanofi’s biologics unit Genzyme has been granted approval for a new drug to treat the rare genetic disorder Gaucher’s disease.

Cerdelga (eliglustat) is now available for the long-term treatment of patients with the Type 1 form of Gaucher’s disease, which affects around 10,000 people worldwide and 6,000 in the US.

Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase, and is a rare genetic condition. 

The enzyme deficiency causes fatty materials to collect in the spleen, liver and bone marrow. The major signs of Gaucher’s disease include liver and spleen enlargement, low red blood cell counts (anaemia), low blood platelet counts and bone problems.

Despite being for such a small population, Sanofi already markets Cerezyme (imiglucerase) for the disease, which it acquired via its 2011 purchase of Genzyme.

But both medicines will face competition from Pfizer’s new treatment Elelyso (taliglucerase alfa), which was approved for the same licence as Sanofi’s drugs in May this year.

Elelyso is slightly different from these drugs as it is the established FDA-approved plant cell-based ERT for Gaucher disease – as the other two use animal-based ERT.

And there is a fourth treatment on the market in the shape of Shire’s Vpriv (velaglucerase alfa), a drug which brought in $256 million last year for the firm.

Shire has been buoyed by Genzyme’s manufacturing issues that have been ongoing since 2009, something which has slowed production of its drug. It still managed to produce $914 million in sales for 2013 however, and holds the majority market share for Gaucher’s disease.

Cerdelga is a hard gelatin capsule containing eliglustat that is taken as a pill. In patients with Gaucher disease Type 1, the drug slows down the production of the fatty materials by inhibiting the metabolic process that forms them.

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 than infusion.

It is unclear how many Gaucher patients on intravenous drugs will make the switch to Cerdelga, Genzyme’s chief executive Dr David Meeker told journalists before the approval. He added the company plans to price its new drug on a par with Cerezyme.

The two drugs also work differently as Cerezyme is an enzyme replacement therapy and accounts for about 70% of the Gaucher market. The new drug works by reducing the amount of fatty lipids produced by the body.

Cerdelga may generate $749 million in sales in 2020, according to analysts’ estimates produced by Bloomberg.

Price wars

Pfizer said that its drug will cost 25% less than Cerezyme, a move the company hopes will help it seize market share from its rivals. Vpriv is currently priced at around 15% less than Cerezyme, which has an average annual cost of about $300,000 (£180,500).

Ben Adams 

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