The results of the vyndaqel and vyndamax (tafamidis) trials and of recent trials of genetic based therapies for ATTR amyloidosis (patisiran and inotersen) are definitely very encouraging and do give hope for ATTR amyloidosis patients.
On the basis of the results from the recent tafamidis trial, the U.S, the Food and Drug Administration (FDA) have approved tafamidis for cardiomyopathy caused by ATTR amyloidosis although it is not approved for ATTR polyneuropathy in the US.
Tafamidis is approved in Europe for treatment of hereditary ATTR amyloidosis patients with neuropathy but it is not currently available within the NHS, and approval in the EU of tafamidis for cardiomyopathy is expected shortly. It will need to be evaluated by NICE before it can become available within the NHS.
The genetic therapies patisiran and inotersen trials showed that these drugs have efficacy in treating patients with ATTR amyloidosis and neuropathy. They have both been approved in Europe and in the US for treating neuropathy in ATTR amyloidosis and are currently being appraised by NICE for use within the NHS.