New Drug Approved in the EU for Treating Hereditary Transthyretin Amyloidosis

According to a story from, two companies, Ionis Pharmaceuticals, Inc., and its affiliate Akcea Pharmaceuticals, recently announced that its product inotersen (to be marketed as TEGSEDI) has gained approval for marketing authorization from the European Commission. Inotersen has been approved for the treatment of stage 1 and 2 polyneuropathy in adults with hereditary transthyretin amyloidosis (hATTR). The approval was recommended by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use.

About Amyloidosis

Amyloidosis is a group of diseases in which an abnormal protein, called amyloid fibrils, begins to accumulate in body tissues. There are nearly thirty types of amyloidosis, all distinguishing by the misfolding of a specific protein. They can have acquired or genetic causes. Hereditary transthyretin amyloidosis is generally found in people who have a family history of the disease and a history of heart problems and idiopathic neuropathies. Symptoms can include enlarged spleen, swollen legs, diarrhea, bleeding, feeling faint upon standing, enlarged tongue, fatigue, numbness, and weight loss. Hereditary transthyretin amyloidosis often appears in old age. Treatments vary depending on the type, but in this type a liver transplant can cure the disease. Survival rates vary as well, but people with hereditary transthyretin amyloidosis can survive for more than a decade, which is better compared to other types. To learn more about amyloidosis, click here.

A New Opportunity

The decision brings a new option to the table for patients that are facing hATTR, which can be a debilitating and ultimately fatal disease. Inotersen also can be self-administered, meaning that patients will not have to travel in order to receive treatment. This results in improved quality of life for patients. This is also the first drug from Akcea to gain approval, and the launch of the product will also include support services for caregivers and patients.

About Inotersen

Inotersen works by inhibiting the production of the TTR protein. The abnormal formation of this protein is what causes TTR amyloid to begin to build up in the patient’s body. The new drug does carry some risk for serious side effects, such as glomerulonephritis and thrombocytopenia. Because of this, close monitoring of patients is necessary when treatment begins in order to identify these events if they appear. Overall, inotersen offers an exciting new treatment option for patients with hereditary transthyretin amyloidosis.

Check out our partners, the Amyloidosis Foundation!

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