Important Safety Information: Contraindications: CERDELGA is contraindicated in the following patients based on CYP2D6 metabolizer status due to the risk of cardiac arrhythmias from prolongation of the PR, QTc, and/or QRS cardiac intervals: Extensive Metabolizers (EMs) taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or... View more

Cerdelga Overview

Cerdelga is the ONLY first-line oral therapy for most adults with Gaucher disease type 1, indicated for the long-term treatment of both naive and switch patients.1,2

View Indications and Usage

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Cerdelga is indicated for the long-term treatment of most adult patients with Gaucher disease type 1.1 Cerdelga, a substrate reduction therapy (SRT), works by reducing the amount of GL-1 produced, allowing the cells’ residual enzyme activity to break down the substrate.1,4

A noninvasive, oral prescription therapy
A noninvasive, oral prescription therapy1
Studied for long-term use in patients who have never been on ERT and in those switched from other ERTs
Studied for long-term use in patients who have never been on ERT and in those switched from other ERTs1,4,5
Patients can switch to Cerdelga in as little as 24 hours after their last ERT infusion
Patients can switch to Cerdelga in as little as 24 hours after their last ERT infusion1
Most common adverse events (AEs) (≥10%): fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain
Most common adverse events (AEs) (≥10%): fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain1

Cerdelga is supported by the largest clinical trial program in Gaucher disease, with ~400 patients enrolled in 29 countries.6,7

The safety and efficacy of Cerdelga have been demonstrated in clinical trials for both Naive (ENGAGE and Phase 2) and Switch (ENCORE) Patients.1,4,5

A Cerdelga Patient’s Treatment Journey

Hear from Shauna—a real Cerdelga patient—as she shares her experience of living with Gaucher disease type 1 and how, with the help of her health care team, she was able to switch from an ERT to Cerdelga.

References:
  1. Cerdelga [prescribing information]. Cambridge, MA: Sanofi Genzyme. 2021.
  2. Pleat R, Cox TM, Burrow TA, et al. Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trial. Mol Genet Metab Rep. 2016;9:25-28.
  3. Mistry PK, Lukina E, Ben Turkia H, et al. Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: the phase 3 ENGAGE trial. Am J Hematol. 2017;92(11):1170–1176
  4. Mistry PK, Lukina E, Ben Turkia H, et al. Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1. The ENGAGE randomized clinical trial. JAMA. 2015;313(7):695-706.
  5. Cox TM, Drelichman G, Cravo R, et al. Eliglustat compared with imiglucerase in patients with Gaucher’s disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomized, open-label, non-inferiority trial. Lancet. 2015;385(9985):2355-2362.
  6. Data on file. Sanofi Genzyme
  7. Peterschmitt MJ, Cox GF, Ibrahim J, et al. A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency, timing, and duration. Blood Cells Mol Dis. 2018;68:185-191.
  8. Cox TM, Drelichman G, Cravo R, et al. Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy. Blood. 2017;129(17):2375-2383.
  9. Lukina E, Watman N, Dragosky M, et al. Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: final results from the phase 2 trial. AM J Hematol. 2019;94(1):29-38.
  10. Lau H, Lukina E, Watman N, et al. Long-term treatment response based on severity of Gaucher disease type 1 at baseline after 8 years of treatment with oral eliglustat: final efficacy and safety results from a phase 2 clinical trial in treatment-naïve adults. Oral presentation at: WORLDSymposium™; February 5-9, 2018; San Diego.