y mass index; mNIS, modified Neuropathy Impairment Score; QoL-DN,Quality of Life – Diabetic Neuropathy; SD, standard deviation; SEM, standard error of the mean a All endpoints analyzed using the mixed-effect model repeated measures (MMRM) method. b A lower value indicates less impairment/fewer symptoms. c A higher number indicates less disability/less impairment.d mBMI: body mass index (BMI; kg/m2) multiplied by serum albumin (g/L); a higher number indicates better nutritional status.
A decrease in Norfolk QoL-DN score indicates improvement.
A decrease in mNIS+7 indicates improvement.
indicates between-group treatment difference, shown as the LS mean difference (95% CI) for ONPATTRO – placebo.
mNIS+7 change scores are rounded to the nearest whole number; last available post-baseline scores were used.
Categories are mutually exclusive; patients who died before 18 months are summarized in the “Death” category only.
indicates between-group treatment difference, shown as the LS mean difference (95% CI) for ONPATTRO – placebo.
Categories are mutually exclusive; patients who died before 18 months are summarized in the “Death” category only.
Patients receiving ONPATTRO experienced similar improvements relative to placebo in mNIS+7 and Norfolk QoL-DN
score across all subgroups including age, sex, race, region, NIS score, Val30Met mutation status, and disease stage.
16 HOW SUPPLIED/STORAGE AND HANDLING
16.1 How Supplied
ONPATTRO is a sterile, preservative-free, white to off-white, opalescent, homogeneous solution for intravenous infusion
supplied as a 10 mg/5 mL (2 mg/mL) solution in a single-dose glass vial. The vial stopper is not made with natural rubber
latex. ONPATTRO is available in cartons containing one single-dose vial each.
The NDC is: 71336-1000-1.
16.2 Storage and Handling
Store at 2°C to 8°C (36°F to 46°F). Do not freeze. Discard vial if it has been frozen.
If refrigeration is not available, ONPATTRO can be stored at room temperature up to 25°C (up to 77°F) for up to 14 days.
For storage conditions of ONPATTRO after dilution in the infusion bag, see Dosage and Administration (2.3).
17 PATIENT COUNSELING INFORMATION
Infusion-Related Reactions
Inform patients about the signs and symptoms of infusion-related reactions (e.g., flushing, dyspnea, chest pain, rash,increased heart rate, facial edema). Advise patients to contact their healthcare provider immediately if they experiencesigns and symptoms of infusion-related reactions [see Warnings and Precautions (5.1)].
Recommended Vitamin A SupplementationInform patients that ONPATTRO treatment leads to a decrease in vitamin A levels measured in the serum. Instruct
patients to take the recommended daily allowance of vitamin A. Advise patients to contact their healthcare provider ifthey experience ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness) and refer them to anophthalmologist if they develop these symptoms [see Warnings and Precautions (5.2)].
PregnancyInstruct patients that if they are pregnant or plan to become pregnant while taking ONPATTRO they should inform theirhealthcare provider. Advise female patients of childbearing potential of the potential risk to the fetus [see Use in SpecialPopulations (8.1)].
Manufactured for: Alnylam Pharmaceuticals, Inc.
300 Third Street, Cambridge, MA 02142By: |
