APOLLO—the largest controlled study in hereditary transthyretin-mediated (hATTR) amyloidosis1

Global, randomized, double-blind, multicenter, placebo-controlled study of 225 patients1-3

Population 225 hATTR amyloidosis patients with polyneuropathya
2:1 Randomization
2:1 Randomization
0.3 mg/kg
IV q3w


IV q3w

Primary Endpoint

Change in mNIS+7, a composite measure of neuropathy impairment, from baseline at 18 months

Key Secondary Endpointb

Norfolk QoL-DN

Select Additional Endpointsb

  • Serum TTR
  • COMPASS 31
  • R-ODS
  • NIS-W
  • 10MWT
  • mBMI
  • EQ-5D-5L

93% of patients treated with ONPATTRO and 62% of patients treated with placebo completed 18 months of the assigned treatment.3

aStudy patients had a diagnosis of hATTR amyloidosis with polyneuropathy caused by any transthyretin (TTR) variant, had FAP stage I or II disease, had a Neuropathy Impairment Score (NIS) of 5-130, and were permitted to have previously used tetramer stabilizers.2
bEndpoints assessed at 18 months.

10MWT=10-meter walk test; COMPASS 31=Composite Autonomic Symptom Score 31; EQ-5D-5L=EuroQoL 5 Dimensions 5 Levels; FAP=familial amyloid polyneuropathy; mBMI=modified body mass index; mNIS+7=modified Neuropathy Impairment Score + 7; NIS-W=Neuropathy Impairment Score-Weakness; Norfolk QoL-DN=Norfolk Quality of Life-Diabetic Neuropathy; R-ODS=Rasch-built Overall Disability Scale.

When patients completed their participation in the 18-month APOLLO study, 99%c of eligible patients elected to enter the global OLE study.2
c186 of 187 patients.

The APOLLO study included a diverse patient population
  • Global study1,2

    225 patients from 19 countries
  • Broad age range2

    24 to 83 years old (median 62 years)
  • Multiple variants2,3

    39 different TTR variants were represented in the study population
  • Prior treatment2

    53% of patients had previously been treated with tafamidis or diflunisal
Disease characteristics of patient population1,3,d


Stage 1 polyneuropathy3
Unimpaired ambulation
(mostly mild sensory, motor, and autonomic neuropathy in lower limbs)


Stage 2 polyneuropathy3
Assistance with ambulation required
(mostly moderate impairment progression to the lower limbs, upper limbs, and trunk)

dFAP stage percentages do not add up to 100% due to inclusion of one stage 3 patient.2

Hear from a physician about ONPATTRO

Take an in-depth look at the ONPATTRO clinical results with Dr. Marcus Urey, a physician with experience managing hATTR amyloidosis.

Important Safety Information and Indication

Important Safety Information

Infusion-Related Reactions

Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO® (patisiran). In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.

To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.

Reduced Serum Vitamin A Levels and Recommended Supplementation

ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).

Adverse Reactions

The most common adverse reactions that occurred in patients treated with ONPATTRO were upper respiratory tract infections (29%) and infusion-related reactions (19%).


ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

For additional information about ONPATTRO, please see the full Prescribing Information.


1. Adams D, Suhr OB, Dyck PJ, et al. BMC Neurol. 2017;17(1):181.

2. Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. N Engl J Med. 2018;379(1):11-21.

3. ONPATTRO Precribing Information. Cambridge, MA: Alnylam Pharmaceuticals, Inc.