Patisiran Brand Name– ONPATTRO
What is Patisiran
Patisiran is a parenteral small interfering ribonucleic acid (siRNA) indicated for the treatment of peripheral nerve disease (polyneuropathy) due to hereditary transthyretin-mediated amyloidosis (hATTR) in adults.
In a randomized, placebo-controlled trial, patisiran-treated patients experienced significantly greater improvements in the modified Neuropathy Impairment Score +7 (mNIS+7) and the Quality of Life-Diabetic Neuropathy (QoL-DN) total score after 18 months of treatment compared to patients who received placebo.
Improvements in these scores reflect gains in physiological outcomes (such as muscle strength, reflexes, sensation) and functional outcomes (such as walking, nutritional status, and performance of activities of daily living) with patisiran treatment.
Infusion-related adverse reactions are common with patisiran; premedication is necessary for all patients
Indications
- amyloidosis-associated polyneuropathy
For the treatment of hereditary transthyretin amyloidosis-associated polyneuropathy
Side Effects
- abdominal pain
- antibody formation
- arthralgia
- AV block
- back pain
- blurred vision
- chest pain (unspecified)
- chills
- cough
- dizziness
- dyspepsia
- dyspnea
- edema
- erythema
- fatigue
- flushing
- headache
- hypertension
- hypotension
- infection
- infusion-related reactions
- injection site reaction
- muscle cramps
- musculoskeletal pain
- nausea
- palpitations
- pharyngitis
- phlebitis
- rash
- rhinitis
- sinusitis
- syncope
- vertigo
- xerophthalmia
Monitoring Parameters
- laboratory monitoring not necessary
Contraindications
- breast-feeding
- infusion-related reactions
- pregnancy
- vitamin A deficiency
Interactions
There are no drug interactions associated with Patisiran products.