One additional patient had pre-existing anti-drug antibodies.

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For patients weighing 100 kg or more, the recommended dosage is 30 mg.

Onpattro treatment leads to a decrease in vitamin A levels in the blood, and vitamin A is essential for normal fetal development, however, excessive levels of vitamin A are associated with adverse developmental effects.

Tell your caregiver if you feel warm, dizzy, nauseated, light-headed, or have a headache, stomach pain, chest pain, or trouble breathing.

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 2.

Forty-three percent of patients had Val30Met mutations in the transthyretin gene; the remaining patients had 38 other point mutations.

Sixty-two percent of ONPATTRO-treated patients had non-Val30Met mutations, compared to 48 of the placebo-treated patients.

One patient (0.7) discontinued ONPATTRO because of an infusion-related reaction.

Table 1 lists the adverse reactions that occurred in at least 5 of patients in the ONPATTRO-treated group and that occurred at least 3 more frequently than in the placebo-treated group in the randomized controlled clinical trial.

Table 1: Adverse Reactions from the Placebo-Controlled Trial that Occurred in at Least 5 of ONPATTRO-treated Patients and at Least 3 More Frequently than in Placebo-treated Patients Adverse Reaction ONPATTRO N148 Placebo N77 Upper respiratory tract infections a 29 21 Infusion-related reaction b 19 9 Dyspepsia 8 4 Dyspnea c,d 8 0 Muscle spasms c 8 1 Arthralgia c 7 0 Erythema c 7 3 Bronchitis e 7 3 Vertigo 5 1 a Includes nasopharyngitis, upper respiratory tract infection, respiratory tract infection, pharyngitis, rhinitis, sinusitis, viral upper respiratory tract infection, upper respiratory tract congestion.

Four serious adverse reactions of atrioventricular (AV) heart block (2.7) occurred in ONPATTRO-treated patients, including 3 cases of complete AV block.

Ocular adverse reactions that occurred in 5 or less of ONPATTRO-treated patients in the controlled clinical trial, but in at least 2 of ONPATTRO-treated patients, and more frequently than on placebo, include dry eye (5 vs.

Extravasation was observed in less than 0.5 of infusions in clinical studies, including cases that were reported as serious.

Immunogenicity The detection of antibody formation is highly dependent on the sensitivity and specificity of the assay.

In addition, the observed incidence of antibody (including neutralizing antibody) positivity in an assay may be influenced by several factors, including assay methodology, sample handling, timing of sample collection, concomitant medications, and underlying disease.

For these reasons, comparison of the incidence of antibodies to ONPATTRO in the studies described below with the incidence of antibodies in other studies or to other products may be misleading.

Anti-drug antibodies to ONPATTRO were evaluated by measuring antibodies specific to PEG2000-C-DMG, a lipid component exposed on the surface of ONPATTRO.

In the placebo-controlled and open-label clinical studies, 7 of 194 (3.6) patients with hATTR amyloidosis developed anti-drug antibodies during treatment with ONPATTRO.

Dmg-Peg