Genzyme extends Gaucher disease options in Europe

The European Commission (EC) has granted marketing authorisation for Genzyme's orphan drug Cerdelga (eliglustat), a first-line oral therapy for adults living with Gaucher disease type 1 (GD1).

The drug was licensed in the US in August 2014 and brings an effective new capsule option for those with Gaucher disease. Genzyme also sells leading injectable enzyme replacement therapy, Cerezyme for the same condition, and will continue to market both.

Cerdelga has a different mode of action, being a ceramide analogue inhibitor of glucosylceramide synthase. It works by reducing the production of glucosylceramide, which builds up in the cells and tissues of people with GD1. It is indicated for those with GD1 who are CYP2D6 poor metabolisers, intermediate metabolisers or extensive metabolisers.

This is not the first pill developed for Gaucher disease; Actelion's Zavesca was launched in the US in 2003, but only holds a small share of the market as it is generally prescribed for those who cannot have enzyme replacement therapies.

The EC approval means Cerdelga should be rolled out commercially in EU countries from this year. It is also under review by other regulatory authorities around the world.

The drug is unsuitable for a small number of patients who metabolise Cerdelga more quickly or at an undetermined rate. Also, because Cerdelga is predicted to cause mild increases in ECG intervals at substantially elevated plasma concentrations, its use should be avoided in patients with cardiac disease.

The EC approval was based on data from the largest clinical research programme conducted in GD1, with approximately 400 patients treated in 29 countries. In the phase III ENGAGE trial of treatment-naïve patients, improvements were seen across spleen size, platelet levels, haemoglobin levels and liver volume after nine months. The ENCORE phase III trial, which assessed disease stability in patients previously treated with enzyme replacement therapy, met the criteria for non-inferiority to an enzyme replacement therapy (imiglucerase) in spleen volume, haemoglobin levels, platelet counts and liver volume.

Gaucher disease is an inherited condition affecting fewer than 10,000 people worldwide.

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