Efficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan

Table 4 Summary of adverse events (safety population)

	SA-ER	Placebo
	(N = 10)	(N = 4)
	n (%) [Number of Events]	n (%) [Number of Events]
Any adverse events	9 (90.0) [55]	4 (100.0) [17]
Any adverse drug reactions	0 (0.0) [0]	0 (0.0) [0]
Serious adverse events	1 (10.0) [1]	1 (25.0) [1]
Serious adverse drug reactions	0 (0.0) [0]	0 (0.0) [0]
Adverse events leading to death	0 (0.0) [0]	0 (0.0) [0]
Adverse events leading to drug withdrawn	0 (0.0) [0]	0 (0.0) [0]
Adverse events occurring in 2 or more subjects
Eye disorders	2 (20.0) [2]	0 (0.0) [0]
Dry eye	2 (20.0) [2]	0 (0.0) [0]
Gastrointestinal disorders	6 (60.0) [19]	2 (50.0) [5]
Diarrhoea	2 (20.0) [11]	1 (25.0) [1]
Gastrooesophageal reflux disease	2 (20.0) [2]	0 (0.0) [0]
General disorders and administration site conditions	2 (20.0) [2]	2 (50.0) [2]
Pyrexia	2 (20.0) [2]	2 (50.0) [2]
Immune system disorders	3 (30.0) [4]	1 (25.0) [1]
Immunisation reaction	3 (30.0) [4]	1 (25.0) [1]
Infections and infestations	3 (30.0) [5]	2 (50.0) [2]
Nasopharyngitis	2 (20.0) [3]	2 (50.0) [2]

N number of subjects in the treatment group; MedDRA medical dictionary for regulatory activities; n number of subjects with events; SA-ER sialic acid extended-release
Subjects with more than one event within a Preferred Term or System Organ Class are counted once for each category. Adverse events were encoded according to MedDRA Ver. 25.0

ISSN: 1750-1172