How does ONPATTRO® (patisiran) treat the polyneuropathy caused by hereditary ATTR (hATTR) amyloidosis?

Liver and TTR protein.

Transthyretin (TTR) is a protein primarily made in the liver

Liver and less TTR protein.

ONPATTRO decreases the amount of TTR protein made in the liver

Amyloid deposits.

A genetic mutation in the TTR gene causes the TTR protein to form clusters known as 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 different parts of the body, leading to symptoms of hATTR amyloidosis

ONPATTRO® (patisiran) infusion.

ONPATTRO
infusion

Liver and TTR protein.

Transthyretin (TTR) is a
protein
primarily made
in the liver

Liver and less TTR protein.

ONPATTRO decreases the
amount of TTR protein made
in the liver

Amyloid deposits.

A genetic mutation in the TTR gene causes the TTR protein to form clusters known as 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 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

Nerve function was assessed by a scale called mNIS+7 that measured strength in hands, feet, arms, and legs; blood pressure upon standing; reflexes; and response to temperature/vibration.

Quality of life was evaluated by the Norfolk Quality of Life (Norfolk QoL-DN) questionnaire that asked patients about the severity of their symptoms, how often they experienced them, and what impact they had on their daily lives.

During the study, the effects of ONPATTRO on other aspects of the disease, such as disability, nutritional health, and symptoms related to digestion and dizziness, were also assessed.

Wheeler, treated with ONPATTRO®  (patisiran).

After my doctor shared the study results with us, my family and I decided ONPATTRO was the right option for me.

WHEELER, treated with ONPATTRO

Hear from patients treated with ONPATTRO


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

ONPATTRO significantly improved
nerve function and quality of life

NERVE
FUNCTION

QUALITY
OF LIFE

Patients receiving placebo
continued to get worse

NERVE
FUNCTION

QUALITY
OF LIFE

Of patients who received ONPATTRO:

More than half (56%) showed improvement in nerve function from their starting point in the study, compared with 4% of those receiving placebo

 

More than half (51%) showed improvement in quality of life from their starting point in the study, compared with 10% of those receiving placebo

Of patients who received ONPATTRO:

For patients who did not show improvement in nerve function, progression of neuropathy was slowed compared with that in patients who received placebo

ONPATTRO significantly improved
nerve function and quality of life

NERVE
FUNCTION

QUALITY
OF LIFE

Patients receiving placebo
continued to get worse

NERVE
FUNCTION

QUALITY
OF LIFE

Of patients who received ONPATTRO:

More than half (56%) showed improvement in nerve function from their starting point in the study, compared with 4% of those receiving placebo

 

More than half (51%) showed improvement in quality of life from their starting point in the study, compared with 10% of those receiving placebo

 

For patients who did not show improvement in nerve function, progression of neuropathy was slowed compared with that in patients who received placebo

Other improvements seen by patients receiving ONPATTRO during the study

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

Decrease in neuropathy symptoms

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

Better nutritional health

ONPATTRO-treated patients maintained a healthier weight

Increase in walking speed

ONPATTRO-treated patients could walk faster

Decrease in pain

Decreased feelings of anxiety and depression

Gary, treated with ONPATTRO® (patisiran).

GARY, treated with ONPATTRO

During the study of 225 patients, on average, patients receiving ONPATTRO maintained the ability to perform some common daily activities, while it became more difficult for patients on placebo to perform those activities

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:

Take a shower

Walk a full flight of stairs

Turn a key in a lock

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