(307) Long-term Safety and Effectiveness of Taliglucerase-alfa for Gaucher disease (GD): An International, Prospective 10-Year Follow Up Drug Registry Study (TALIAS)

Background: Taliglucerase-alfa (Elelyso) is a pharmacologic lysosomal glycosphingolipid-specific β-glucosidase indicated for long-term enzyme replacement therapy (ERT) in patients with Gaucher disease (GD). Although approved in the US for adults and children 10-12 years ago, limited real-world data on the long-term safety and effectiveness of Elelyso are available.

Objectives: Characterize the safety profile and effectiveness of Elelyso in patients with GD through the solicited collection of data from routine clinical care.

Methods: Taliglucerase-alfa Surveillance (TALIAS) was an international, prospective drug registry established in 2013, sponsored by Pfizer Inc. Data on adverse events and effectiveness endpoints were collected from 24 sites in Albania, Israel, Turkey, and the United States (US). This report presents data from September 2013 to November 2023.

Results: 151 patients (15.9% children < 18 years; 55.6% females) were included. About 53% had at least 5 years and ~22% had 7 years of follow up. Most participants were from Israel (43.1%) followed by US (22.5%), Albania (19.2%) and Turkey (15.2%). Twenty-six (17.2%) were treatment-naïve and 114 (75.4%) had received ERT prior to Elelyso. Hemoglobin levels and platelet counts increased, and liver and spleen volume decreased in the treatment-naïve participants. In those participants who had prior ERT, these parameters remained the same or slightly improved following Elelyso treatment. Three participants (2.0%) experienced infusion-related serious adverse events (AEs) including, chest pain, back pain, shortness of breath, and anaphylaxis. There were 72 (47.7%) all causality treatment-emergent AEs, of which 32 (21.2%) experienced serious AEs. Sixty-two (41.1%) discontinued from the study including: 37 (24.5%) who lost to follow up, 5 (3.3%) were not willing to participate, 7 (4.6%) discontinued because their investigator declined further participation in the study, 1 (0.7%) due to an AE, 2 (1.3%) for unknown reasons and 10 (6.6%) died. None of the deaths were judged related to Elelyso by the treating physicians.

Five women enrolled in the pregnancy-lactation sub-study and had 6 uneventful live birth deliveries. All these participants continued Elelyso during their pregnancies and while lactating. One newborn had a cleft palate (palatoschisis), which the investigator and Sponsor concurred was most likely unrelated to Elelyso.

Conclusions: No unexpected safety issues were identified in this long-term follow-up study. Study data are consistent with the known safety and effectiveness profile of Elelyso reported in the pivotal clinical trials.