NAGLAZYME (Page 2 of 5)

5.6 Spinal or Cervical Cord Compression

Spinal or cervical cord compression (SCC) with resultant myelopathy is a known and serious complication of MPS VI. SCC is expected to occur in the natural history of the disease, including in patients on NAGLAZYME. There have been post-marketing reports of patients treated with NAGLAZYME who experienced the onset or worsening of SCC requiring decompression surgery. Patients with MPS VI should be monitored for signs and symptoms of spinal/cervical cord compression (including back pain, paralysis of limbs below the level of compression, urinary and fecal incontinence) and given appropriate clinical care.

6 ADVERSE REACTIONS

Serious and or clinically significant adverse reactions described elsewhere in labeling include:

  • Anaphylaxis and Hypersensitivity Reactions [see Warnings and Precautions (5.1)]
  • Immune-Mediated Reactions [see Warnings and Precautions (5.2)]
  • Risk of Acute Cardiorespiratory Failure [see Warnings and Precautions (5.3)]
  • Acute Respiratory Complications Associated with Administration [see Warnings and Precautions (5.4)]
  • Infusion Reactions [see Warnings and Precautions (5.5)]
  • Spinal or Cervical Cord Compression [see Warnings and Precautions (5.6)]

6.1 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 observed in the clinical trials of another drug and may not reflect the rates observed in clinical practice.

NAGLAZYME was studied in a randomized, double-blind, placebo-controlled trial in which 19 patients received weekly infusions of 1 mg/kg NAGLAZYME and 20 patients received placebo; of the 39 patients 66% were female, and 62% were White, non-Hispanic. Patients were aged 5 years to 29 years. NAGLAZYME-treated patients were approximately 3 years older than placebo-treated patients (mean age 13.7 years versus 10.7 years, respectively).

Serious adverse reactions experienced in this trial include apnea, pyrexia, and respiratory distress. Severe adverse reactions include chest pain, dyspnea, laryngeal edema, and conjunctivitis. The most common adverse reactions requiring interventions were infusion reactions .

Table 1 summarizes the adverse reactions that occurred in the placebo-controlled trial in at least 2 patients more in the NAGLAZYME‑treated group than in the placebo-treated group.

Table 1: Adverse Reactions that Occurred in the Placebo-Controlled Trial in at least 2 Patients More in the NAGLAZYME Group than in the Placebo Group
MedDRA Preferred Term NAGLAZYME (n = 19) Placebo (n = 20 * )
No. Patients (%) No. Patients (%)
*
One of the 20 patients in the placebo group dropped out after Week 4 infusion
All 19 (100) 20 (100)
Abdominal Pain 9 (47) 7 (35)
Ear Pain 8 (42) 4 (20)
Arthralgia 8 (42) 5 (25)
Pain 6 (32) 1 (5)
Conjunctivitis 4 (21) 0
Dyspnea 4 (21) 2 (10)
Rash 4 (21) 2 (10)
Chills 4 (21) 0
Chest Pain 3 (16) 1 (5)
Pharyngitis 2 (11) 0
Areflexia 2 (11) 0
Corneal Opacity 2 (11) 0
Gastroenteritis 2 (11) 0
Hypertension 2 (11) 0
Malaise 2 (11) 0
Nasal Congestion 2 (11) 0
Umbilical Hernia 2 (11) 0
Hearing Impairment 2 (11) 0

Four open-label clinical trials were conducted in MPS VI patients aged 3 months to 29 years with NAGLAZYME administered at doses of 0.2 mg/kg (n = 2), 1 mg/kg (n = 55), and 2 mg/kg (n = 2). The mean exposure to the recommended dose of NAGLAZYME (1 mg/kg) was 138 weeks (range = 54 to 261 weeks). Two infants (12.1 months and 12.7 months) were exposed to 2 mg/kg of NAGLAZYME for 105 and 81 weeks, respectively.

In addition to those listed in Table 1, common adverse reactions observed in the open-label trials include pruritus, urticaria, pyrexia, headache, nausea, and vomiting. The most common adverse reactions requiring interventions were infusion reactions. Serious adverse reactions included laryngeal edema, urticaria, angioedema, and other hypersensitivity reactions. Severe adverse reactions included urticaria, rash, and abdominal pain.

Observed adverse events in four open-label studies (up to 261 weeks treatment) were not different in nature or severity to those observed in the placebo-controlled study. No patients discontinued during open-label treatment with NAGLAZYME due to adverse events.

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