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Clinical Trials Experience

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

The most common adverse reactions to CERDELGA (occurring in ≥ 10% of the 126 GD1 patients treated with CERDELGA across Trials 1 and 2) were fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.

The adverse reaction profile of CERDELGA is based on two controlled studies, Trials 1 and 2. Table 1 presents the profile from the 9-month double-blind, randomized, placebocontrolled trial of 40 treatment-naïve patients (Trial 1). Patients were between the ages of 16 and 63 on the date of the first dose of study drug, and included 20 males and 20 females.

Table 1: Adverse Reactions Occurring in ≥ 10% of Treatment-Naïve GD1 Patients and More Frequently than Placebo (Trial 1)

Adverse Reaction CERDELGA
(N=20)
Placebo
(N=20)
Patients
n (%)
Patients
n (%)
Arthralgia 9 ( 45) 2 ( 10)
Headache 8 ( 40) 6 ( 30)
Migraine 2 ( 10) 0 ( 0)
Flatulence 2 ( 10) 1 ( 5)
Nausea 2 ( 10) 1 ( 5)
Oropharyngeal pain 2 ( 10) 1 ( 5)

Table 2 presents the profile from the 12-month open-label, randomized, imiglucerasecontrolled trial of 159 treated patients switching from enzyme replacement therapy (ERT) (Trial 2). Patients were between the ages of 18 and 69 on the date of the first dose of CERDELGA, and included 87 females and 72 males.

Table 2: Adverse Reactions Occurring in ≥ 5% of GD1 Patients Switching from Enzyme Replacement Therapy to CERDELGA and More Frequently than Imiglucerase (Trial 2)*

Adverse Reaction CERDELGA (N=106)
Patients n (%)
Imiglucerase (N=53)
Patients n (%)
Fatigue 15 ( 14) 1 ( 2)
Headache 14 ( 13) 1 ( 2)
Nausea 13 ( 12) 0 ( 0)
Diarrhea 13 ( 12) 2 ( 4)
Back pain 13 ( 12) 3 ( 6)
Pain in extremity 12 ( 11) 1 ( 2)
Upper abdominal pain 11 ( 10) 0 ( 0)
Dizziness 9 ( 8) 0 ( 0)
Asthenia 9 ( 8) 0 ( 0)
Cough 7 ( 7) 2 ( 4)
Dyspepsia 7 ( 7) 1 ( 2)
Gastroesophageal reflux disease 7 ( 7) 0 ( 0)
Constipation 5 ( 5) 0 ( 0)
Palpitations 5 ( 5) 0 ( 0)
Rash 5 ( 5) 0 ( 0)
*Trial 2 was not designed to support comparative claims for CERDELGA for the adverse reactions reported in this table.

In an uncontrolled study, with up to 4 years of treatment, in 26 patients, the types and incidences of adverse reactions were similar to Trials 1 and 2.