Table 3 Overview of treatment-emergent adverse events during treatment with olipudase alfa (1 year of treatment for those in the former placebo group crossing over to olipudase alfa and 2 years of treatment for the original olipudase alfa group)

Any treatment-emergent adverse event

35 (100)

584

Severity

 Mild

34 (97.1)

447 (76.5)

 Moderate

26 (74.3)

125 (25.9)

 Severe

6 (17.1)

11 (1.9)

Any serious treatment-emergent adverse events

10 (28.6)

13 (2.2)

Any treatment-emergent adverse events potentially related to study drug

24 (68.6)

151 (25.9)

Any serious treatment-emergent adverse events potentially related to study drug

1 (2.9)

1 (0.2)

Headache

10 (28.6)

59

53/59

47 mild, 11 moderate

Alanine aminotransferase increased

4 (11.4)

11

1/11

6 mild, 4 moderate, 1 severe

Aspartate aminotransferase increased

4 (11.4)

6

1/6

3 mild, 3 moderate

Urticaria

3 (8.6)

5

5/5

3 mild, 2 moderate

Vomiting

3 (8.6)

4

4/4

All mild

Abdominal pain

3 (8.6)

3

1/3

2 mild, 1 moderate

Nausea

3 (8.6)

3

3/3

2 mild, 1 moderate

Pyrexia

3 (8.6)

3

2/3

2 mild, 1 moderate

Pruritis

2 (5.7)

2

2/2

1 mild, 1 moderate

Joint swelling

2 (5.7)

5

4/5

All mild

Chills

2 (5.7)

2

0/2

All mild

Blood bilirubin increased

2 (5.7)

2

0/2

All mild

Dyspnea

2 (5.7)

2

1/2

All mild

Erythema

2 (5.7)

2

2/2

All mild

Musculoskeletal chest pain

2 (5.7)

2

0/2

All mild