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Cerdelga, first-line oral therapy approved by FDA for the treatment of adults with Gaucher disease type 1


The FDA ( Food and Drug Administration ) has approved Cerdelga ( Eliglustat ) capsules, the only first-line oral therapy for certain adult Gaucher disease type 1 patients. A small number of adult patients who metabolize Cerdelga more quickly or at an undetermined rate, as detected by an established genetic laboratory test, will not be eligible for Cerdelga treatment.

The FDA approval was based on efficacy data from two positive phase 3 studies for Cerdelga: one in patients new to therapy ( Trial 1 ), and the other in patients switching from approved enzyme replacement therapies ( Trial 2 ).
The filing also incorporated four years of efficacy data from the Cerdelga phase 2 study.

In Trial 1, improvements were seen across the following endpoints after 9 months on Cerdelga: spleen size, platelet levels, hemoglobin levels, and liver volume.
Patients continue to receive Cerdelga in the extension period, and the majority of patients have been on treatment for over eighteen months.

Trial 2 met the pre-specified criteria for non-inferiority to an enzyme replacement therapy ( Imiglucerase ), which was a composite endpoint of each of the following parameters: spleen volume, hemoglobin levels, platelet counts, and liver volume. Patients continue to receive Cerdelga in the extension period, and the majority of patients have been on treatment for over two years.

The most common adverse reactions ( greater than or equal to 10% ) are fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.

Eliglustat is a specific ceramide analogue inhibitor of glucosylceramide synthase ( IC50 = 10 ng/mL ) with broad tissue distribution. It reduces the production of glucosylceramide, the substance that builds up in the cells and tissues of people with Gaucher disease.

Gaucher disease is an inherited condition affecting fewer than 10,000 people worldwide. People with Gaucher disease do not have enough of an enzyme, beta-glucosidase ( glucocerebrosidase ) that breaks down a certain type of fat molecule. As a result, lipid engorged cells amass in different parts of the body, primarily the spleen, liver and bone marrow.
Accumulation of Gaucher cells may cause spleen and liver enlargement, anemia, excessive bleeding and bruising, bone disease and a number of other signs and symptoms.
The most common form of Gaucher disease, type 1, generally does not affect the brain.

Source: Genzyme, 2014

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