Two approved treatments for Gaucher disease type 1 patients — Cerdelga (eliglustat) and Cerezyme (imiglucerase) — are equally effective as treatments, working increase hemoglobin and platelets levels, and reduce spleen and liver size, according to a small review study.
The research, “The Clinical Efficacy of Imiglucerase versus Eliglustat in Patients with Gaucher’s Disease Type 1: A Systematic Review,” was published in the Journal of Research in Pharmacy Practice.
Cerdelga and Cerezyme, both marketed by Sanofi Genzyme, are treatment options type 1 patients. While Cerezyme is an enzyme replacement therapy (ERT) providing an analog of beta-glucocerebrosidase — defective in these people — Cerdelga is a substrate reduction therapy (SRT) that lessens the amount of the fat molecule glucosylceramide.
Oral administration and more effective crossing of the blood-brain barrier are among the advantages of SRTs. In contrast, ERTs — the first approved treatments for Gaucher type1 — require lifelong intravenous administration and can be more costly. (Cerdelga was approved by the U.S. Food and Drug Administration in 2014; Cerezyme was approved in 1994.)
Researchers at Tehran University of Medical Sciences in Iran conducted a systematic review of the literature comparing the efficacy of Cerezyme to that of Cerdelga in Gaucher type 1 patients.
Out of 1,619 studies considered, only three — conducted between 2015 and 2016 involving a total of 311 patients — met their requirements. Two were randomized clinical trials, with doses used that ranged from 15 to 130 U/kg for Cerezyme, and 50 to 150 mg twice daily for Cerdelga.
The studies’ primary goals were changes in the levels of hemoglobin (the protein that carries oxygen in the blood), platelet counts, and liver and spleen size, all of which can be abnormal in Gaucher type 1 patients. Secondary outcomes were the immunological adverse events of the therapies and bone complications.
Results showed that the two treatments led to similar increases in hemoglobin level and platelet count, as well as to equal lessening of size in the liver and spleen.
Bone is often affected in Gaucher patients, but the study found that bone mineral density kept within the normal range after 12 months of treatment with either Cerdelga or Cerezyme. Each medication also helped to maintain disease stability, and in a similar manner.
The most frequent adverse events were diarrhea, joint pain, fatigue, and headache. Cerdelga did not induce a significant effect on heart rate or cardiac repolarization — a change in voltage involved in heart beats. Regarding depolarization, the other type of changed cardiac voltage, Cerdelga had only a slightly greater effect than Cerezyme. No adverse events were considered related to treatment and none resulted in study withdrawal.
“The findings of this review showed that both medicines are effective in the treatment of GD [Gaucher disease] Type 1 and there is no statistically significant difference between their efficacies,” the scientists wrote.
However, the investigators cautioned against drawing definitive conclusions, given the low number of randomized controlled comparisons of the two treatments, small sample sizes, short follow-up periods, and inadequate therapeutic outcome measures.
They recommended further studies to determine if Cerdelga is as effective as ERTs in patients with severe disease, whether it is safe in people with nerve damage, and its possible advantages in preventing long-term complications and associated conditions.