.
Table 4: Adverse Reactions Reported in More Than 10% of Pediatric Patients Receiving CRYSVITA in Studies 1 and 2
Adverse Reaction
Study 1
(N=52)
n (%) Study 2
(N=13)
n (%) Overall
(N=65)
n (%)
Headache 38 (73) 1 (8) 39 (60)
Injection site reaction1 35 (67) 3 (23) 38 (59)
Vomiting 25 (48) 6 (46) 31 (48)
Pyrexia 23 (44) 8 (62) 31 (48)
Pain in extremity 24 (46) 3 (23) 27 (42)
Vitamin D decreased2 19 (37) 2 (15) 21 (32)
Rash3 14 (27) 1 (8) 15 (23)
Toothache 12 (23) 2 (15) 14 (22)
Myalgia 9 (17) 1 (8) 10 (15)
Tooth abscess 8 (15) 3 (23) 11 (17)
Dizziness4
8 (15) 0 (0) 8 (12)
n = number of patients with an event; N = total number of patients who received at least one dose of CRYSVITA
1 Injection site reaction includes: injection site reaction, injection site erythema, injection site pruritus, injection site swelling, injection site pain, injection site rash, injection site bruising, injection site discoloration, injection site discomfort, injection site hematoma, injection site hemorrhage, injection site induration, injection site macule, and injection site urticaria
2 Vitamin D decreased includes: vitamin D deficiency, blood 25-hydroxycholecalciferol decreased, and vitamin D decreased
3 Rash includes: rash, rash pruritic, rash maculopapular, and rash pustular
4 Dizziness includes: dizziness, and dizziness exertional
Hypersensitivity Reactions
In pediatric patients, the most frequent potential hypersensitivity events were rash (22%), injection site rash (6%), and urticaria (5%).
Hyperphosphatemia
In pediatric studies, there were no events of hyperphosphatemia reported.
Injection Site Reactions (ISR)
In pediatric studies, approximately 58% of the patients had a local reaction (e.g. injection site urticaria, erythema, rash, swelling, bruising, pain, pruritus, and hematoma) at the site of CRYSVITA injection. Injection site reactions were generally mild in severity, occurred within 1 day of injection, lasted approximately 1 to 3 days, required no treatment, and resolved in almost all instances.
Adverse Reactions in Adult Patients with XLH
The safety data described below reflect exposure to CRYSVITA in 68 adult XLH patients, age 20-63 years (mean age 41 years), of whom most were white/Caucasian (81%) and female (65%). These patients were enrolled in a randomized, double-blind, placebo-controlled Phase 3 study in adults with XLH (Study 3: CRYSVITA = 68, Placebo = 66), in which patients received CRYSVITA at a mean dose of 0.95 mg/kg (range 0.3 – 1.2 mg/kg) subcutaneously every 4 weeks at Week 24. Adverse reactions reported in more than 5% of CRYSVITA-treated patients and 2 patients or more than with placebo from the 24-week placebo-controlled portion of Study 3 are shown in Table 5.
Table 5:Adverse Reactions Occurring in More Than 5% of CRYSVITA-Treated Adult Patients and in at Least 2 Patients More Than with Placebo in Study 3
Adverse Reaction CRYSVITA
(N=68)
n (%) Placebo
(N=66)
n (%)
Back pain 10 (15) 6 (9)
Headache1 9 (13) 6 (9)
Tooth infection2 9 (13) 6 (9)
Restless legs syndrome 8 (12) 5 (8)
Vitamin D decreased3 8 (12) 3 (5)
Dizz |
