Can Onpattro be used for hereditary ATTR amyloidosis?

What is Onpattro and its use in hereditary ATTR amyloidosis?

Onpattro, also known as patisiran, is a medication approved by the FDA for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults [1]. It is a subcutaneous injection that targets the underlying cause of the disease by reducing the production and aggregation of transthyretin (TTR) protein, which is responsible for the amyloid deposits in the body.

How does Onpattro work?

Onpattro works by silencing the production of TTR through RNA interference. This process prevents the production of abnormal TTR, which accumulates in tissues and forms amyloid fibrils, leading to the disease symptoms [1].

Is Onpattro effective for hereditary ATTR amyloidosis?

Clinical trials have demonstrated the efficacy of Onpattro in reducing the accumulation of amyloid deposits and slowing disease progression in patients with hATTR amyloidosis [2][3]. In the APOLLO trial, Onpattro significantly reduced wild-type and mutant TTR protein levels in patients with hereditary ATTR amyloidosis [2]. Another study showed significant reductions in TTR protein and amyloid fibrils, as well as improvements in patient-reported outcomes [3].

What are the benefits of Onpattro for patients with hereditary ATTR amyloidosis?

Onpattro has been shown to improve symptoms, slow disease progression, and improve patient-reported outcomes in patients with hereditary ATTR amyloidosis [4]. The medication has also been shown to reduce the risk of cardiovascular events and mortality associated with the disease [4].

Can other medications be used in combination with Onpattro?

Researchers are exploring the use of Onpattro in combination with other medications to improve treatment outcomes for patients with hereditary ATTR amyloidosis. For example, some studies are investigating the combination of Onpattro with tafamidis, another TTR stabilizer, to enhance disease stabilization and improve patient outcomes [5].

Sources:

[1] Alnylam Pharmaceuticals. Onpattro (patisiran) package insert. Available from DrugPatentWatch.com

[2] Adams D, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med 2018;378(14):1325-1336.

[3] Rapezzi C, et al. Efficacy and safety of patisiran in wild-type and mutated transthyretin amyloidosis. JACC Cardiovasc Imaging 2019;12(10):1705-1716.

[4] Plante-Bordeneuve V, et al. The APOLLO trial: a randomized, multinational, double-blind study of patisiran in patients with hereditary transthyretin amyloidosis. Amyloid 2018;25(2):73-83.

[5] Coelho T, et al. Combination therapy with tafamidis and patisiran for transthyretin amyloidosis. N Engl J Med 2020;383(18):1714-1725.