How does ONPATTRO® treat the polyneuropathy caused by hATTR amyloidosis?

Liver and TTR protein.

TTR is a protein made mostly in the liver

Liver and less TTR protein.

ONPATTRO decreases the amount of TTR protein made in the liver

Amyloid deposits.

A change in the TTR gene causes the TTR protein to cluster and form amyloid deposits

Fewer amyloid deposits.

This helps to decrease the amount of amyloid deposits and may result in fewer symptoms

Amyloid deposits build up in the body

Amyloid deposits build up in different parts of the body, leading to symptoms of hATTR amyloidosis

ONPATTRO® (patisiran) infusion.

ONPATTRO
infusion

Liver and TTR protein.

TTR is a protein made mostly in the liver

Liver and less TTR protein.

ONPATTRO decreases the
amount of TTR protein made
in the liver

Amyloid deposits.

A change in the TTR gene causes the TTR protein to cluster and form amyloid deposits

Fewer amyloid deposits.

This helps to decrease the amount of amyloid deposits and may result in fewer symptoms

Amyloid deposits build up in the body

Amyloid deposits build up in different parts of the body, leading to symptoms of hATTR amyloidosis

ONPATTRO® (patisiran) infusion.

ONPATTRO
infusion

Did you know

TTR protein was reduced by an average of 84% after 18 months in a study of adults with polyneuropathy caused by hATTR amyloidosis who were treated with ONPATTRO.


How ONPATTRO was studied

The safety and effectiveness of ONPATTRO were evaluated in an 18-month study of 225 adult patients with polyneuropathy caused by hATTR amyloidosis.

During the study, some patients received ONPATTRO and their results were compared with those of patients who received a placebo.

The clinical study focused on changes in nerve function and quality of life at 18 months

Study on changes in nerve function at 18 months

Nerve function was assessed using a scale called mNIS+7, which measured strength and sensation in the hands, feet, arms, and legs; reflexes; and blood pressure upon standing.

Study on changes in quality of life at 18 months

Quality of life was evaluated using the Norfolk QoL-DN questionnaire, which asked patients about the severity of their polyneuropathy symptoms, how often they experienced them, and what impact they felt they had on their daily lives.

Effects of ONPATTRO on walking, nutritional health, and ability to perform activities

The effects of ONPATTRO on other aspects of the disease, including walking ability, nutritional health, and ability to perform activities of daily living, were also evaluated.

From the start of the study to 18 months, nerve function and quality of life significantly improved for patients who received ONPATTRO compared with those who received placebo

ONPATTRO significantly improved
nerve function and quality of life

NERVE
FUNCTION

QUALITY
OF LIFE

Patients who received
placebo got worse

NERVE
FUNCTION

QUALITY
OF LIFE

At 18 months:

56% of patients who received ONPATTRO regained some nerve function from the start of treatment, compared with 4% of those who received placebo

 

51% of patients who received ONPATTRO reported better quality of life from the start of treatment compared with 10% of those who received placebo

At 18 months:

For patients who received ONPATTRO and did not regain some nerve function, progression of their neuropathy was slowed compared with those who received placebo

ONPATTRO significantly improved
nerve function and quality of life

NERVE
FUNCTION

QUALITY
OF LIFE

Patients who received
placebo got worse

NERVE
FUNCTION

QUALITY
OF LIFE

At 18 months:

56% of patients who received ONPATTRO regained some nerve function from the start of treatment, compared with 4% of those who received placebo

 

51% of patients who received ONPATTRO reported better quality of life from the start of treatment compared with 10% of those who received placebo

 

For patients who received ONPATTRO and did not regain some nerve function, progression of their neuropathy was slowed compared with those who received placebo

Other improvements seen by patients who received ONPATTRO during the study

After 18 months of treatment, when compared to patients who received placebo, patients who received ONPATTRO experienced:

Decreases in neuropathy symptoms using ONPATTRO

Decrease in neuropathy symptoms

Including diarrhea, constipation, dizziness, difficulty urinating, and vomiting

Better nutritional health using ONPATTRO

Better nutritional health

Patients treated with ONPATTRO maintained a healthier weight

Increases in walking speed with ONPATTRO

Increase in walking speed

Patients treated with ONPATTRO maintained a better walking speed

Decrease in pain with ONPATTRO

Decrease in pain

Decreased feelings of anxiety and depression with ONPATTRO

Decreased feelings of anxiety and depression

During the study of 225 patients, on average, patients who received ONPATTRO were better able to perform common daily activities compared with those who received placebo

This was assessed by a 24-item questionnaire called R-ODS that asked patients to rate their ability to complete tasks both at the beginning of the study and after 18 months of treatment. Some of these tasks included the ability to:

Rating ability to take a shower using ONPATTRO

Take a shower

Rating ability to walk up a flight of stairs using ONPATTRO

Walk a full flight of stairs

Rating ability to turn a key in a lock using ONPATTRO

Turn a key in a lock

Rating ability to do the dishes using ONPATTRO

Do the dishes

Damage to nerves that affects sensation, movement, strength, and bodily functions such as digestion, urination, and sexual function.

A treatment without any active medication.

The 24-item questionnaire asked patients about their ability to:

  • Eat
  • Make a sandwich
  • Brush teeth
  • Wash upper body
  • Wash lower body
  • Sit on a toilet
  • Dress upper body
  • Go to the general practitioner
  • Read a newspaper or book
  • Go shopping
  • Travel by public transportation
  • Move a chair
  • Catch an object
  • Bend and pick up an object
  • Carry and put down a heavy object
  • Walk and avoid obstacles
  • Walk outside
  • Run
  • Dance
  • Stand for hours
  • Take a shower
  • Walk a full flight of stairs
  • Turn a key in a lock
  • Do the dishes