According to a story on pharmaphorum.com, the UK’s National Institute for Health and Care Excellence (NICE) has given a positive opinion for the drug vutrisiran (marketed as Amvuttra) as a treatment for hereditary ATTR amyloidosis, a rare disease. This opinion will allow for the therapy to be covered on the NHS for England and Wales. Developed by Alnylam Pharmaceuticals, this therapy follows the company’s earlier treatment for the disease called patisiran (marketed as Onpattro), which received approval in 2019.
About Hereditary ATTR Amyloidosis
Hereditary ATTR Amyloidosis is a disease which is characterized by the buildup of amyloid fibrils in body tissue. Amyloid fibrils are a type of abnormal protein. The disease is caused by a heritable genetic mutation or can be acquired. Symptoms of the illness include swelling, heart failure, irregular heartbeat, shortness of breath, fatigue, weight loss, easy bruising and bleeding, stroke, lung problems, enlarged liver, and changes in skin color. Treatment approaches for hereditary ATTR amyloidosis includes liver transplant; This can actually cure the disease, but the procedure carries substantial risks and side effects. This form of amyloidosis has a better prognosis than some of the more common types with some patients surviving for over a decade. It is estimated to affect around 50,000 people worldwide. To learn more about hereditary ATTR amyloidosis, click here.
A Treatment with Clear Advantages
The principal advantage of Amvuttra over earlier amyloidosis therapies like Onpattro is the easier dosing regimen. While Onpattro is delivered as an intravenous infusion every several weeks, Amvuttra can be administered under the skin every three months. The draft guidance released by NICE appears to indicate that use of Amvuttra would have cost saving benefits and a reduced treatment burden on patients.
The therapy inhibits the production of amyloid protein, a mechanism that can significantly slow down the progression of the disease symptoms, which can develop into serious polyneuropathy, leaving patients with loss of feeling in the legs and hands.
Overall, it looks like Amvuttra has the potential to be a real game changer for people living with hereditary ATTR amyloidosis in the UK.