Treatment with Cerdelga (eliglustat) leads to clinically meaningful long-term improvements in hemoglobin and platelet levels in patients with Gaucher disease type 1 (GD1), data from long-term Phase 2 and Phase 3 ENGAGE trials show.
The results were recently presented at the 59th American Society of Hematology (ASH) Annual Meeting & Exposition, Dec. 9-12 in Atlanta, Georgia, in a presentation titled “Long-Term Hematologic Response to Oral Eliglustat in Treatment-naive Patients with Gaucher Disease Type 1: Final Results from a Phase 2 and the Phase 3 Engage Trials.”
Cerdelga, a substrate reduction therapy (SRT), halts the accumulation of the fat molecule glucosylceramide (the hallmark of GD) by partially inhibiting the glucosylceramide synthase enzyme – the one responsible for the production of glucosylceramide.
The U.S. Food and Drug Administration (FDA) approved Cerdelga as a treatment for Gaucher disease type 1 in 2014, a decision supported by Phase 3 trials, including ENGAGE.
In the Phase 3 ENGAGE trial (NCT00891202), patients were randomized to a placebo or Cerdelga. After nine months of treatment, Cerdelga-treated patients met the primary endpoint – a significant reduction in spleen and liver volumes – and secondary endpoints – increases in hemoglobin concentration and platelet count (vital for blood clotting). In contrast, placebo-treated patients became slightly worse. The results were maintained during the trial’s extension phase.
Fourteen patients enrolled in an open-label extension study and received Cerdelga for 4.5 years. The data showed that Cerdelga maintains its efficacy, with hemoglobin level increasing – from 12.0 to 13.4 g/dL – and mean platelet count increases showing a boost of 87%.
The study, “Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1: The ENGAGE Randomized Clinical Trial,” was published in the journal JAMA.
In the Phase 2 trial (NCT00358150), patients showed improvements in spleen volume, hemoglobin level, and platelet counts. Data from 19 patients treated with Cerdelga for eight years continued to show improvements in hemoglobin levels – from 11.3 to 13.4 g/dL – and increases of 110% in platelet counts.
The phase 2 trial results were published in the journal Blood in a study titled “A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1.”
Overall, “in previously untreated patients with GD1, clinically meaningful improvements in hematologic parameters continued or were maintained over time after 8 years of treatment with eliglustat in the Phase 2 trial and after 4.5 years of eliglustat in the Phase 3 ENGAGE trial,” the study concluded.