Cerdelga Seen to Improve Hemoglobin, Platelet Levels in Gaucher Type 1 Adults, Trials Show

Cerdelga Seen to Improve Hemoglobin, Platelet Levels in Gaucher Type 1 Adults, Trials Show

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.

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.
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Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.

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