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Label indication ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. ONPATTRO contains a transthyretin-directed small interfering RNA and is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. FDA label
Also known as patisiran (ALN-TTR02), ONPATTRO, ALN-TTR02, patisiran-lipid nanoparticle (LNP)
patisiran · 8 trials · 15 indications
Distance in meters walked in 6 minutes, longer distances indicate greater functional capacity. Missing 6MWT values due to non-COVID-19 death or inability to walk due to ATTR disease progression were imputed using the worst 10th percentile change observed in the DB period. Missing 6-MWT values due to other reasons are multiply imputed to create 100 complete datasets. The change from baseline is averaged across the 100 complete datasets.
Serum TTR was assessed using enzyme linked immunosorbent assay (ELISA). The average of the percentage reduction in serum TTR observed at Month 6 and at Month 12 is first calculated for each patient and then the median (95% CI) of these averaged values is summarized for the Safety Analysis Set.
The mNIS+7 is a composite score that measures neurologic impairment which includes the following components: physical exam of lower limbs, upper limbs and cranial nerves to assess motor strength/weakness, electrophysiologic measurement of small and large nerve fiber function, sensory testing and postural blood pressure. The mNIS+7 is scored from 0 (no impairment) to 304 points (maximum impairment). A higher score indicates a worse outcome.
AE is any untoward medical occurrence in a participant or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in mNIS+7 at 18 months. The mNIS+7 is a composite score that quantitates motor, sensory, and autonomic neurologic impairment due to injury of large and small nerves. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.
The number of participants experiencing at least one adverse event (AE), at least one serious adverse event (SAE) and study drug discontinuation (due to any reason).
to assess the severity of functional impairment of motor and sensory nerves.NIS is a measure of motor strength, comprised of cranial nerve and both upper and lower limb motor assessments. The minimum and maximum values are 0 and 192, respectively. A higher score indicates a worse outcome.
Change in Norfolk Quality of Life Questionnaire (Norfolk QOL-DN).The change from baseline in Norfolk QoL-DN at 24 months. The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome.
Composite Autonomic Symptom Score (COMPASS) 31a self-assessment instrument for patient reported autonomic symptoms such as dizziness, constipation, diarrhea, nausea/vomiting, and incontinence. The minimum and maximum values are 0 and 100, respectively. A higher score indicates a worse outcome
| Arm | Type | Description |
|---|---|---|
| Patisiran | EXPERIMENTAL | Participants will be administered multiple doses of patisiran in the double-blind and open-label extension period. |
| Placebo | PLACEBO_COMPARATOR | Participants will be administered multiple doses of placebo in the double-blind period. In the open-label extension period, participants will be administered multiple doses of patisiran. |
| Vutrisiran + Vutrisiran (HELIOS-A) | EXPERIMENTAL | Participants will receive vutrisiran 25 mg subcutaneous (SC) injection once every 3 months (q3M) for 18 months during the Treatment Period followed by vutrisiran 50 mg SC injection once every 6 months (q6M) or vutrisiran 25 mg q3M during the Randomized Treatment Extension (RTE) Period. Upon implementation of Amendment 6, participants receiving vutrisiran SC 50 mg q6M will transition to vutrisiran SC 25 mg q3M at their next scheduled dosing. |
| Patisiran + Vutrisiran (HELIOS-A) | ACTIVE_COMPARATOR | Participants will receive patisiran 0.3 mg/kg intravenous (IV) infusion once every 3 weeks (q3w) for 18 months during the Treatment Period followed by vutrisiran 50 mg SC injection once q6M or vutrisiran 25 mg q3M during the RTE Period. Upon implementation of Amendment 6, participants receiving vutrisiran SC 50 mg q6M will transition to vutrisiran SC 25 mg q3M at their next scheduled dosing. |
| Prior Placebo Group of Study 004 | EXPERIMENTAL | Participants who received placebo and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 milligrams per kilogram (mg/kg) patisiran intravenously (IV) once every 3 weeks (Q3W) up to 65.5 months. |
| Prior Patisiran Group of Study 004 | EXPERIMENTAL | Participants who received patisiran and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 66.9 months. |
| Prior Patisiran Group of Study 003 | EXPERIMENTAL | Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
| patisiran (ALN-TTR02) | ACTIVE_COMPARATOR | - |
| Sterile Normal Saline (0.9% NaCl) | PLACEBO_COMPARATOR | - |
| open label | OTHER | single open arm label |
| Name | Type | Description |
|---|---|---|
| Placebo | DRUG | Normal saline (0.9% NaCl) matching volume of patisiran doses will be administered intravenously. |
| Patisiran | DRUG | Patisiran will be administered by intravenous (IV) infusion. |
| Vutrisiran | DRUG | Vutrisiran will be administered by SC injection. |
| patisiran (ALN-TTR02) | DRUG | administered by intravenous (IV) infusion |
| Sterile Normal Saline (0.9% NaCl) | DRUG | administered by intravenous (IV) infusion |
Inclusion Criteria: * Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hereditary ATTR amyloidosis with cardiomyopathy or wild-type ATTR amyloidosis with cardiomyopathy * Medical history of heart failure with at least 1 prior hospitalization for heart failure, or c...
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|---|---|---|---|---|
| AstraZeneca PLC | AZN | 8 | PHASE3 | CAEL-101, AZD0120, GC012F |
| Intellia Therapeutics, Inc. | NTLA | 1 | PHASE3 | Nexiguran ziclumeran |
| Immix Biopharma, Inc. | IMMX | 2 | PHASE3 | NXC-201 |
| Johnson & Johnson | JNJ | 3 | PHASE2 | Daratumumab, Bortezomib, Teclistamab, JNJ-79635322 |
| BridgeBio Pharma, Inc. | BBIO | 2 | PHASE3 | Acoramidis |
| GSK plc Sponsored ADR | GSK | 1 | PHASE2 | Belantamab mafodotin |
| Alnylam Pharmaceuticals, Inc | ALNY | 4 | EARLY_PHASE1 | patisiran |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE2 | Isatuximab |
| AbbVie, Inc. | ABBV | 1 | PHASE1 | ABBV-383 |
| Regeneron Pharmaceuticals, Inc. | REGN | 1 | PHASE1 | linvoseltamab |
Patisiran is an RNA therapy developed by Alnylam Pharmaceuticals, Inc. (ticker ALNY) for transthyretin (TTR)-mediated amyloidosis, a rare disease. It is designed to reduce production of the TTR protein that forms amyloid deposits in nerves and other tissues. It is also known as patisiran (ALN-TTR02).
Patisiran is used for transthyretin (TTR)-mediated amyloidosis, including hereditary and familial forms, wild-type ATTR amyloidosis, and associated polyneuropathies. It is also studied in ATTR amyloidosis with cardiomyopathy. It is an RNA therapy in the rare disease area.
Patisiran targets transthyretin (TTR). It is an RNAi inhibitor, meaning it is designed to interfere with the production of the TTR protein. By reducing TTR, the therapy aims to lower the protein that misfolds and forms amyloid deposits in tissues such as peripheral nerves and the heart.
Patisiran is developed by Alnylam Pharmaceuticals, Inc., which trades under the ticker ALNY. Alnylam is the company responsible for the development of this RNA therapy for transthyretin-mediated amyloidosis.
Patisiran is in Phase 3 development. It is an investigational RNA therapy and is not described as approved. Clinical development includes completed Phase 3 studies and an early Phase 1 study that is active but not recruiting.
Patisiran has been studied in trials including NCT03997383 (APOLLO-B, Phase 3, 360 participants with ATTR amyloidosis with cardiomyopathy), NCT03862807 (Phase 3, 24 participants with hereditary ATTR amyloidosis after liver transplant), and NCT05023889 (early Phase 1, 10 participants with wild-type ATTR amyloidosis and polyneuropathies).
Yes, patisiran is also known as ALN-TTR02. ALN-TTR02 is an alternative name for the same investigational RNA therapy developed by Alnylam Pharmaceuticals, Inc. for transthyretin (TTR)-mediated amyloidosis.