PARIS (dpa-AFX) - Genzyme, a Sanofi company (SNYNF.PK, SNY) announced that it has received approval from the U.S. Food and Drug Administration or FDA for its Cerdelga capsules, the only first-line oral therapy for certain adult Gaucher disease type 1 patients.
The company said 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. Cerdelga is expected to be available to patients within a month.
Genzyme has been researching an oral therapy for Gaucher disease for fifteen years, from early chemistry and preclinical research through clinical development. The Cerdelga clinical development program is the largest ever conducted in Gaucher disease, with approximately 400 patients treated in 29 countries.
Genzyme noted that 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, the company stated.
Genzyme said Trial 2 met the pre-specified criteria for non-inferiority to an enzyme replacement therapy, which was a composite endpoint of each of the following parameters: spleen volume, hemoglobin levels, platelet counts, and liver volume.
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