Date: 2012-02-08
Type of information: Results
phase: 2
Announcement: results
Company: Genzyme (USA - MA), a Sanofi company (France)
Product: eliglustat tartrate
Action
mechanism: specific inhibitor of glucosylceramide synthase
Disease: Gaucher\'s disease
Therapeutic area: Rare diseases - Genetic diseases
Country:
Trial details:
Latest
news: Genzyme has announced four-year follow-up data from patients enrolled in the phase 2 clinical trial for its investigational oral therapy for Gaucher disease type 1, eliglustat tartrate. Sustained or further improvements were observed across all endpoints, including markers of bone disease, at the four-year timepoint. The results will be presented for the first time this week at the Lysosomal Disease Network WORLD Symposium in San Diego, Calif.
Genzyme previously reported that the 52-week phase 2 trial of eliglustat tartrate had met its primary composite endpoint: a clinically meaningful response in at least two of three endpoints (improvements in spleen size, hemoglobin and platelet levels) in individual patients. Patients have continued to receive eliglustat tartrate in the extension portion of the study for over four years.
The data from patients on eliglustat tartrate after four years indicate continued or stabilized improvements across all endpoints:
• Spleen and liver volumes decreased from baseline by a mean of 63 percent and 28 percent respectively.
• Hemoglobin and platelet levels had increased from baseline by a mean of 2.3 g/dL and 95 percent respectively.
• All patients (100 percent) had met at least three of the four hematologic and visceral therapeutic goals established for enzyme replacement therapy.
• These data also indicate continued improvement in bone mineral density by DXA, with a mean T-score increase of 0.8 from baseline in the lumbar spine.
In the phase 2 study, the most common adverse events reported in greater than two patients through four years of treatment included viral infections (six patients), urinary tract and upper respiratory tract infections (four patients each), and nasopharyngitis, sinusitis, arthralgia, pain in extremity, headache, increased blood pressure, abnormal nerve conduction study, abdominal pain, and diarrhea (three patients each). Ten drug-related adverse events, including one serious event, were reported in eight patients. All related events were mild in severity.
