According to a story from Pharmafield, The UK’s National Institute for Health and Care Excellence (NICE) has recently approved a first in class gene silencing therapy for coverage on the country’s NHS. This therapy will treat hereditary transthyretin mediated (hATTR) amyloidosis, a rare disease. The drug is called patisiran (marketed as ONPATTRO). This announcement is big news for this patient community, which suffered from a lack of covered treatments.
Amyloidosis is a group of diseases which are characterized by the build up amyloid fibrils in body tissue. Amyloid fibrils are a type of abnormal protein. As there are several different types of amyloidosis, the cause can vary. Some forms are acquired while others are linked to genetic factors. One of the genetic forms is hATTR amyloidosis. Symptoms of the disease 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 amyloidosis includes chemotherapy and stem cell transplant; other options vary depending on the type of amyloidosis. Prognosis varies depending on the type; hATTR amyloidosis has a more favorable prognosis compared to other types, with some patients surviving for more than ten years. To learn more about amyloidosis, click here.
Treating HATTR Amyloidosis With Patisiran
The approval of patisiran also gives UK patients the opportunity to access a treatment that helps address the underlying mechanism of amyloidosis and reduces the production of the abnormal amyloid proteins. Studies have shown that the drug has the potential to halt the progression of symptoms and even reverse some of the worst effects for many patients with hATTR amyloidosis. The drug definitely has the potential to improve survival and quality of life. As a gene silencing therapy, patisiran utilizes RNAi in order to halt the production of the majority of the faulty, disease causing protein.
The coverage of the therapy on the NHS was the result of almost a year of negotiations between NICE and Alynam Pharmaceuticals, the company that developed and manufactures patisiran.