Amvuttra (vutrisiran) has received European Medicines Agency (EMA) approval. The EMA's Committee for Medicinal Products for Human Use (CHMP) recommended the marketing authorization of Amvuttra for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients [1]. This recommendation was based on positive results from the Phase 3 APOLLO study, which demonstrated Amvuttra's efficacy in reducing the levels of mutant and wild-type transthyretin protein [1][2].
What is Amvuttra used for?
Amvuttra is indicated for the treatment of hATTR amyloidosis in adult patients. This condition is a rare, progressive, and debilitating disease caused by the misfolding of transthyretin (TTR) protein, leading to amyloid deposition in nerves, the heart, and other organs [1][2]. Amvuttra targets the root cause of hATTR amyloidosis by reducing the production of TTR protein in the liver [1].
How does Amvuttra work?
Amvuttra is an RNA interference (RNAi) therapeutic. It utilizes a small interfering RNA (siRNA) to silence the TTR gene in the liver, thereby decreasing the amount of TTR protein produced [1][2]. This reduction in TTR protein aims to prevent or reverse the accumulation of amyloid deposits that cause disease symptoms [1].
What are the clinical trial results for Amvuttra?
The EMA's recommendation for Amvuttra was supported by data from the APOLLO study. This Phase 3 trial evaluated Amvuttra in adult patients with hATTR amyloidosis with polyneuropathy. The study met its primary efficacy endpoint, showing a significant reduction in serum TTR levels compared to placebo at 18 months. Amvuttra also demonstrated improvements in polyneuropathy and quality of life assessments [1][2].
When might Amvuttra be available in Europe?
Following the CHMP's positive opinion, the European Commission will review the recommendation. A final decision on whether to grant marketing authorization for Amvuttra is expected in the coming months. If approved, Amvuttra will become available to eligible patients in European Union member states [1].
What is the difference between Amvuttra and Onpattro?
Amvuttra and Onpattro (patisiran) are both RNAi therapeutics approved for hATTR amyloidosis. However, they differ in their chemical structure and delivery. Amvuttra is an N-acetylgalactosamine (GalNAc)-conjugated siRNA, which allows for subcutaneous administration. Onpattro is also an siRNA therapeutic but is administered intravenously [2]. Both drugs target TTR protein production but are distinct molecules.
What are potential side effects of Amvuttra?
While specific side effect profiles will be detailed in the product's label upon approval, common adverse reactions observed in clinical trials for similar RNAi therapies may include injection site reactions, upper respiratory tract infections, and nausea [2]. Patients should discuss any concerns about side effects with their healthcare provider.
Sources:
[1] https://www.drugpatentwatch.com/news/ema-recommends-amvuttra-vutrisiran-for-hattr-amyloidosis
[2] https://www.drugpatentwatch.com/news/ambry-genetics-receives-fda-approval-for-amvuttra-vutrisiran-to-treat-hattr-amyloidosis-polyneuropathy