In short
Patisiran is an investigational drug being studied in clinical trials for the treatment of transthyretin-mediated (ATTR) amyloidosis, a rare and serious condition caused by the buildup of abnormal proteins in various organs and tissues. This article summarizes key findings from clinical trials evaluating patisiran's safety and effectiveness in patients with different forms of ATTR amyloidosis, including those with polyneuropathy and cardiomyopathy.
At a glance
- Drug Name
- Patisiran (ALN-TTR02, ONPATTRO)
- Mechanism of Action
- RNA interference (RNAi) therapeutic targeting TTR gene
- Administration
- Intravenous infusion, typically every 3 weeks
- Target Conditions
- Hereditary ATTR amyloidosis with polyneuropathy, ATTR amyloidosis with cardiomyopathy, wild-type ATTR amyloidosis
- Key Efficacy Measures
- mNIS+7, Norfolk QoL-DN, 6MWT, KCCQ-OS, NT-proBNP, echocardiogram parameters
- Safety Monitoring
- Adverse events, serious adverse events, study discontinuations
- Notable Findings
- Improvements in neurological impairment, quality of life, and walking ability; reduction in serum TTR levels
- Ongoing Research
- Long-term safety and efficacy, alternative dosing regimens, expanded patient populations
What is Patisiran?
Patisiran is a medication used to treat certain types of amyloidosis, a rare and serious condition. It is also known by its brand name ONPATTRO or its research code ALN-TTR02. Patisiran is part of a new class of drugs that work by interfering with the production of specific proteins in the body.
What Conditions Does Patisiran Treat?
Patisiran is primarily used to treat the following conditions:
- Hereditary Transthyretin-mediated (ATTRv) Amyloidosis: This is an inherited condition where abnormal proteins build up in various organs, causing damage.
- Polyneuropathy: This is nerve damage that occurs as part of ATTRv amyloidosis.
- Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy): This is a form of amyloidosis that affects the heart.
How Does Patisiran Work?
Patisiran works by reducing the production of a protein called transthyretin (TTR) in the liver. In patients with ATTRv amyloidosis, this protein is abnormal and builds up in various parts of the body, causing damage. By decreasing the amount of TTR produced, patisiran helps to reduce the accumulation of these harmful protein deposits.
How is Patisiran Administered?
Patisiran is given as an intravenous (IV) infusion, which means it's delivered directly into your bloodstream through a vein. The typical dosing schedule is:
- Once every 3 weeks (q3w)
- The dose is usually 0.3 milligrams per kilogram (mg/kg) of body weight
- For patients weighing 100 kg or more, the maximum dose is 30 mg
Before receiving patisiran, patients are typically given premedications to reduce the risk of infusion-related reactions.
Efficacy of Patisiran
Clinical trials have shown that patisiran can be effective in treating the symptoms of ATTRv amyloidosis. Some of the benefits observed include:
- Improved neurological function: Measured by the Neuropathy Impairment Score (NIS), which assesses muscle weakness, sensation, and reflexes.
- Better quality of life: As measured by the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) questionnaire.
- Improved disability scores: Using the Rasch-Built Overall Disability Scale (R-ODS).
- Reduced autonomic symptoms: Measured by the Composite Autonomic Symptom Score (COMPASS-31).
- Improved nutritional status: As indicated by the modified Body Mass Index (mBMI).
Safety and Side Effects
Like all medications, patisiran can cause side effects. The most common side effects reported in clinical trials include:
- Infusion-related reactions
- Upper respiratory tract infections
- Headache
- Diarrhea
It's important to note that not everyone experiences side effects, and your doctor will monitor you closely during treatment.
Ongoing Research
Patisiran continues to be studied in various clinical trials to further understand its benefits and potential uses. Some areas of ongoing research include:
- Long-term safety and efficacy in patients with ATTRv amyloidosis
- Use in patients who have had liver transplants
- Effectiveness in treating ATTR amyloidosis with cardiomyopathy
- Comparison with other treatments for ATTRv amyloidosis
These ongoing studies will help researchers and doctors better understand how to use patisiran most effectively and safely in different patient populations.