Skip to main content
University of Michigan Innovation Partnerships
University of Michigan Innovation Partnerships

Twice-a-day Cerdelga is slightly better than once-a-day regimen for Gaucher, trial shows


Two Cerdelga (eliglustat) pills a day are slightly better than one pill a day of the same strength at helping Gaucher type 1 patients maintain a stable disease, a Phase 3 clinical trial shows.

The therapy’s maker, Genzyme, conducted the trial, which involved only patients whose disease had become stable while they were taking Cerdelga twice a day.

The report, “Once- versus twice-daily dosing of eliglustat in adults with Gaucher disease type 1: The Phase 3, randomized, double-blind EDGE trial,” was published in the journal Molecular Genetics and Metabolism.

The U.S., the European Union, and a number of other countries have approved Cerdelga.

It is a long-term treatment for the 90 percent of adults with Gaucher disease type 1 who have difficulty metabolizing the liver enzyme CYP2D6.

Genzyme said the oral medicine inhibits the enzyme glucosylceramide synthase. The enzyme produces glucosylceramide, a product of metabolism that accumulates to toxic levels in the tissue and organs of people with Gaucher.

The Phase 3 EDGE trial (NCT01074944) compared the effectiveness and safety of once-a-day and twice-a-day doses of Cerdelga in adults with Gaucher disease type 1. It also compared the doses’ pharmacokinetics — or how the body absorbs, distributes, metabolizes and clears a drug.

Only patients who achieved a stable disease on twice-a-day doses of Cerelga over six to 18 months could participate in the study. Researchers defined stability as patients achieving stable levels of blood hemoglobin, blood platelets, and spleen and liver sizes, and having stable bone symptoms.

Researchers randomly sorted the 131 participants into two groups. One received 100 or 200 mg of Cerelga once a day. The other received 50 or 100 mg doses twice a day.

After one year, 80.4 percent of the once-a-day patients had a stable disease, compared with 83.1 percent of the twice-a-day patients.

The percentages were so close that researchers concluded that “these results confirm the twice-daily dosing recommended for patients” who have difficulty metabolizing CYP2D6.

Both regimens generated similar safety profiles, researchers said. The most frequent adverse events from the treatment were dizziness, dyspepsia, nausea, and headache.

The results showed that “patients on twice-daily eliglustat achieved slightly better clinical stability overall, especially with regard to bone, and this dosing regimen was better tolerated, confirming the dose regimen specified in the drug label for most patients,” the researchers said.

The trial also evaluated Cerdelga’s long-term safety. Patients tolerated it well over the mean treatment period of 3.3 years, researchers said.

This was consistent with findings from the ENCORE Phase 3 trial (NCT00943111). It evaluated Cerdelga’s safety and effectiveness for up to four years in patients who had achieved a stable disease on enzyme replacement therapy.