In 2018 the US Food and Drug Administration (FDA) approved the intravenous medicine, Onpattro (patisiran), created by Alnylam Pharmaceuticals for the treatment of the polyneuropathy (damage affecting the peripheral nerves) of hereditary amyloidosis in adults. Similarly, the European Medicines Agency granted Onpattro (patisiran) a marketing authorisation for adults with Stage 1 and Stage 2 polyneuropathy. Previously, in the European Union there were limited options available to alter the disease progression.
Patients with this type of amyloidosis have a mutation that causes abnormal proteins, amyloids, to accumulate in the body. This leads to weakness, numbness and pain in the extremities. Based on Nobel Prize-winning science on RNA interference, Onpattro (patisiran) is designed to interfere with specific messenger RNA that produce mutated transthyretin (TTR), amyloids, in the liver. This reduces the formation of amyloids and relieves the symptoms of polyneuropathy associated with hATTR amyloidosis. This reduction can slow or stop disease progression. Further information for this medicine's approval can be found here.