Complete adverse effect profile including incidence rates and management
Important Safety Information
This is not a complete list of all possible side effects. Contact your healthcare provider if you experience any unexpected symptoms. For serious or life-threatening side effects, seek emergency medical attention immediately.
6 ADVERSE REACTIONS The following clinically significant adverse reactions are described elsewhere in the labeling: Infusion-Related Reactions [see Warnings and Precautions (5.1) ] The most frequently reported adverse reactions (that occurred in at least 10% of ONPATTRO-treated patients and at least 3% more frequently than on placebo) were upper respiratory tract infections and infusion-related reactions ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Alnylam Pharmaceuticals at 1-877-256-9526 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .
6.1 Clinical Trials Experience Because clinical studies are conducted under widely varying conditions, adverse reaction rates observed in the clinical studies of ONPATTRO cannot be directly compared to rates in the clinical studies of another drug and may not reflect the rates observed in practice.
A total of 224 patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) received ONPATTRO in the placebo-controlled and open-label clinical studies, including 186 patients exposed for at least 1 year, 137 patients exposed for at least 2 years, and 52 patients exposed for at least 3 years.
In the placebo-controlled study, 148 patients received ONPATTRO for up to 18 months (mean exposure 17.7 months).
Baseline demographic and disease characteristics were generally similar between treatment groups.
The median age of study patients was 62 years and 74% were male.
Seventy-two percent of study patients were Caucasian, 23% were Asian, 2% were Black, and 2% were reported as other.
At baseline, 46% of patients were in Stage 1 of the disease and 53% were in Stage
Forty-three percent of patients had Val30Met mutations in the transthyretin gene;
the remaining patients had 38 other point mutations.
5 WARNINGS AND PRECAUTIONS Infusion-related reactions: Monitor for signs and symptoms during infusion.
Slow or interrupt the infusion if clinically indicated.
Discontinue the infusion if a serious or life-threatening infusion-related reaction occurs ( 5.1 ) Reduced serum vitamin A levels and recommended supplementation: Supplement with the recommended daily allowance of vitamin A.
Refer to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur ( 5.2 ) 5.1 Infusion-Related Reactions Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO.
In clinical studies, all patients received premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) to reduce the risk of IRRs.
Like all medications, Onpattro can cause side effects. However, not everyone who takes this medication will experience them. Many side effects are dose-dependent and may improve as your body adjusts to the medication. Others may require dose adjustment or medical attention.
Contact your healthcare provider promptly if you experience:
Seek immediate emergency medical care if you experience signs of: