Sanofi’s Pediatric Eliglustat Trial Completion: What It Means for Gaucher Franchise Growth

Sanofi (SNYNF) announced an update on their ongoing clinical study.

Sanofi Advances Pediatric Gaucher Treatment With Eliglustat Study Completion

Sanofi has completed a late-stage clinical study titled “Open Label, Two Cohort (With and Without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients With Gaucher Disease Type 1 and Type 3.” The study focused on children aged 2 to under 18, aiming first to assess safety and how the body handles the drug, and second to gauge treatment benefit and quality of life. For investors, the significance lies in extending an established Gaucher therapy into younger patients, potentially broadening Sanofi’s rare-disease franchise.

The main treatment tested was eliglustat, an oral drug designed to reduce the build-up of certain fats in cells, which drives Gaucher disease. In one group, children received eliglustat alone. In the other group, they received eliglustat together with imiglucerase, an intravenous enzyme replacement that is already a standard therapy in Gaucher disease, before moving to eliglustat alone if they responded well. The goal was to see if eliglustat can work safely and effectively as a stand‑alone or step‑down option in pediatric care.

The study used an interventional design, meaning doctors actively gave specific treatments and tracked outcomes. Patients were assigned to treatment groups without randomization and followed in sequence, reflecting how therapy might be adjusted in real clinics. There was no blinding, so both doctors and families knew which treatment was given. The main purpose was treatment-focused, looking at safety first, then clinical benefit, rather than simply observing disease progression.

The trial was first submitted in March 2018, marking the formal start of regulatory engagement for this pediatric program. The overall status is listed as completed, indicating that treatment and follow-up have finished and data are in hand. The record was most recently updated on January 12, 2026, signaling that Sanofi has refreshed information and is likely moving toward data analysis, disclosure, and potential label discussions. No results are posted yet, so investors are still waiting for a clear readout on outcomes.

From a market view, this update strengthens Sanofi’s positioning in rare genetic diseases, an area that supports premium pricing and durable cash flows. If results confirm that eliglustat is safe and effective in children, Sanofi could expand use beyond adults, deepen its Gaucher revenue base, and extend the lifecycle of its franchise alongside imiglucerase. This may support sentiment on Sanofi’s long-term growth, especially as investors look for visibility in specialty and rare-disease segments. Competitors in Gaucher, such as Takeda and smaller biotech players working on gene therapies, will watch closely, as a credible oral option in pediatrics could raise the bar for new entrants. Until full data are released, the update is more of a quiet de‑risking step than a major near-term catalyst, but it tilts expectations modestly positive for the Gaucher portfolio.

The study is completed and the record has been recently updated, with further details and future data releases to be made available on the ClinicalTrials portal.

To learn more about SNYNF’s potential, visit the Sanofi drug pipeline page.