Table 3.
AEs in participants with XLH treated with burosumab (safety analysis set).
| MedDRA SOC/PTa,b,c | Burosumab, n = 67 | |
|---|---|---|
| Participants | AEs | |
| Participants with any AE, n (%) | 25 (37.3) | 83 |
| Congenital, familial, and genetic disorders | 1 (1.5) | 1 |
| Craniosynostosis | 1 (1.5) | 1 |
| Ear and labyrinth disorders | 1 (1.5) | 1 |
| Ear pain | 1 (1.5) | 1 |
| Gastrointestinal disorders | 6 (9.0) | 8 |
| Toothache | 2 (3.0) | 3 |
| Abdominal pain | 1 (1.5) | 1 |
| Diarrhea | 1 (1.5) | 1 |
| Infrequent bowel movements | 1 (1.5) | 1 |
| Loose tooth | 1 (1.5) | 1 |
| Vomiting | 1 (1.5) | 1 |
| General disorders and administration site conditions | 7 (10.4) | 11 |
| Pain | 3 (4.5) | 4 |
| Fatigue | 2 (3.0) | 2 |
| Injection site erythema | 2 (3.0) | 2 |
| Chest pain | 1 (1.5) | 1 |
| Injection-site mass | 1 (1.5) | 1 |
| Peripheral swelling | 1 (1.5) | 1 |
| Infections and infestations | 11 (16.4) | 12 |
| Tooth abscess | 7 (10.4) | 7 |
| Abscess jaw | 1 (1.5) | 1 |
| COVID-19 | 1 (1.5) | 1 |
| Gastroenteritis | 1 (1.5) | 1 |
| Viral infection | 1 (1.5) | 1 |
| Viral upper respiratory tract infection | 1 (1.5) | 1 |
| Injury, poisoning, and procedural complications | 2 (3.0) | 3 |
| Accidental poisoning | 1 (1.5) | 1 |
| Head injury | 1 (1.5) | 1 |
| Procedural pain | 1 (1.5) | 1 |
| Investigations | 1 (1.5) | 1 |
| Blood alkaline phosphatase increased | 1 (1.5) | 1 |
| Musculoskeletal and connective tissue disorders | 16 (23.9) | 30 |
| Pain in extremity | 12 (17.9) | 18 |
| Arthralgia | 4 (6.0) | 4 |
| Knee deformity | 2 (3.0) | 2 |
| Limb discomfort | 2 (3.0) | 2 |
| Neck pain | 1 (1.5) | 2 |
| Back pain | 1 (1.5) | 1 |
| Limb deformity | 1 (1.5) | 1 |
| Nervous system disorders | 3 (4.5) | 3 |
| Headache | 2 (3.0) | 2 |
| Restless legs syndrome | 1 (1.5) | 1 |
| Psychiatric disorders | 3 (4.5) | 3 |
| Emotional distress | 1 (1.5) | 1 |
| Irritability | 1 (1.5) | 1 |
| Sleep terror | 1 (1.5) | 1 |
| Respiratory, thoracic, and mediastinal disorders | 2 (3.0) | 2 |
| Asthma | 1 (1.5) | 1 |
| Dyspnea | 1 (1.5) | 1 |
| Skin and subcutaneous tissue disorders | 2 (3.0) | 2 |
| Rash | 2 (3.0) | 2 |
| Uncoded | 6 (9.0) | 6 |
Percentages are calculated using the number of participants in the safety analysis set as denominator.
a
SOC terms ordered alphabetically; PT sorted in order of frequency of the total column within each SOC.
b
A participant can have more than one SOC and more than one PT reported under a given SOC.
c
If a participant experiences the same AE (same SOC or same PT) more than once, participant is only counted once.
AE, adverse event; MedDRA, Medical Dictionary for Regulatory Activities version 23.1; PT, preferred term; SOC, system organ class; XLH, X-linked hypophosphatemia.