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Label indication WAINUA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. WAINUA is a transthyretin-directed antisense oligonucleotide indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. ( 1 ) FDA label
Also known as Eplontersen Solution for Injection
Eplontersen · 3 trials · 3 indications
To evaluate the effect of eplontersen compared to placebo on serum transthyretin (TTR) concentration up to 24 weeks
Change from baseline in Cardiopulmonary exercise test peak VO2 at week 52
To characterize the pharmacokinetic (PK) profiles of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participants
To characterize the pharmacokinetic (PK) profiles of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participants
To characterize the pharmacokinetic (PK) profiles of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participants
To characterize the pharmacokinetic (PK) profiles of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participants
To characterize the pharmacokinetic (PK) profiles of eplontersen following subcutaneous administration of a single 45 mg dose in healthy Chinese participants
| Arm | Type | Description |
|---|---|---|
| Eplontersen | EXPERIMENTAL | Eplontersen by subcutaneous injection once every 4 weeks |
| placebo | PLACEBO_COMPARATOR | Eplontersen-matching placebo by subcutaneous injection once every 4 weeks |
| Eplontersen and ALXN2220 | EXPERIMENTAL | Subcutaneous eplontersen and intravenous ALXN2220 every 4 weeks |
| Eplontersen and placebo | PLACEBO_COMPARATOR | Subcutaneous eplontersen and intravenous placebo every 4 weeks |
| Name | Type | Description |
|---|---|---|
| Eplontersen | DRUG | Eplontersen by subcutaneous injection |
| Placebo | DRUG | Eplontersen-matching placebo by subcutaneous injection |
| ALXN2220 | BIOLOGICAL | ALXN2220 delivered intravenously, once every 4 weeks |
| Eplontersen Solution for Injection | DRUG | an autoinjector with 0.8 mL deliverable volume (at 56 mg/mL concentration) will be provided, total dose is 45 mg. |
Inclusion Criteria: 1. Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements. 2. 20 to 90 years of age (inclusive). 3. Females: must be non-pregnant and non-lactating and either: 1. surgically s...
Eplontersen is an investigational RNA therapy being developed for transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin-mediated amyloidosis. It is designed to reduce transthyretin protein production in patients with these conditions. It is not yet approved and remains in clinical development.
Eplontersen targets transthyretin, also known as TTR. It is an antisense inhibitor, an RNA therapy that binds TTR messenger RNA to reduce production of the transthyretin protein. Lowering TTR protein levels is the intended mechanism in transthyretin amyloid cardiomyopathy and transthyretin-mediated amyloidosis.
Eplontersen is being developed by AstraZeneca PLC, which trades under the ticker AZN. The company is running the clinical development program for the drug across transthyretin amyloid cardiomyopathy and transthyretin-mediated amyloidosis.
Eplontersen is in Phase 3 clinical development. It is investigational and has not been approved by the FDA. The Phase 3 program includes the EPIC-ATTR study in Chinese subjects with transthyretin amyloid cardiomyopathy, and a Phase 2 combination study is also ongoing.
Eplontersen is being studied in NCT06194825, the Phase 3 EPIC-ATTR trial in Chinese subjects with transthyretin amyloid cardiomyopathy, and NCT07608354, a Phase 2 trial combining eplontersen with ALXN2220 versus eplontersen with placebo in ATTR-CM. A completed Phase 1 pharmacokinetic study in healthy Chinese volunteers was registered as NCT06527755.
Yes. Eplontersen solution for injection is an alternative name for eplontersen, the antisense inhibitor targeting transthyretin that AstraZeneca is developing for transthyretin amyloid cardiomyopathy and transthyretin-mediated amyloidosis. Searches for either name refer to the same investigational drug.