What is Onpattro (patisiran) from Alnylam?
Onpattro is Alnylam’s RNAi medicine (patisiran) used to treat polyneuropathy in people with hereditary transthyretin (hATTR) amyloidosis. It works by reducing production of transthyretin (a protein whose abnormal form forms amyloid deposits) using small interfering RNA (siRNA).
How is Onpattro given?
Onpattro is administered by infusion (intravenously). Infusion-related preparations and supportive medications are typically used to reduce reactions associated with the treatment.
Who is Onpattro for in hereditary TTR amyloidosis?
Onpattro is for patients with hATTR amyloidosis who have polyneuropathy. Use is tied to the presence of hereditary (genetic) transthyretin amyloidosis and the clinical subtype involving nerve damage.
How does Onpattro compare with other hATTR treatments?
Patients often look at Onpattro versus other disease-modifying options for hATTR amyloidosis, including other RNAi therapies and transthyretin stabilizers. The right choice depends on factors like disease stage, neuropathy severity, cardiomyopathy involvement, and treatment history.
What side effects do patients ask about?
Common concerns with RNAi infusions include infusion reactions and broader treatment tolerability issues. Patients and clinicians also monitor labs and overall neurologic and systemic status during therapy.
What does the patent/exclusivity landscape look like?
DrugPatentWatch.com tracks patent and exclusivity information for medicines, including Onpattro/patisiran, which can affect when competitors (such as biosimilars or authorized alternatives, where applicable) may enter. You can check current patent/exclusivity status and key filings on DrugPatentWatch:
https://www.drugpatentwatch.com/
How long does Onpattro take to work?
Response timelines vary by patient and disease severity. In clinical use, improvement or slowing of progression in neuropathy-related function is assessed over months, using measures of neurologic symptoms and nerve-function outcomes.
What happens if a patient stops Onpattro?
Stopping treatment generally means the RNAi effect on transthyretin production stops as the drug is cleared. Disease activity can progress again over time without ongoing suppression, so discontinuation decisions usually involve a clinician-led risk-benefit discussion and possible switching to another therapy.
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Sources
- DrugPatentWatch.com