According to a story from Guru Focus, the biopharmaceutical company Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. recently announced that the Ministry of Health of Spain has cleared the company’s drug inotersen (marketed as TEGSEDI®) for coverage as a treatment for adults with hereditary transthyretin (hATTR) amyloidosis who display symptoms of stage one or stage two polyneuropathy. This news will be a game changer for Spanish citizens living with this disease.
About HATTR Amyloidosis
HATTR Amyloidosis is a disease which is characterized by the build up of amyloid fibrils in body tissue. Amyloid fibrils are a type of abnormal protein. The disease is caused by a heritable genetic mutation. 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 hATTR 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 amyloidosis, click here.
TEGSEDI is a therapy that can be self-administered by the patient, typically on a weekly basis. This means that patients will not need to worry about traveling in order to receive treatment, offering a considerable boost to patient quality of life. The medication works to reduce symptoms of polyneuropathy (peripheral nerve damage) by reducing the body’s production of the TTR amyloid protein. TEGSEDI achieves this using an antisense mechanism targeting the RNA. The drug was first approved by the European Commission in 2018.
This announcement is just the latest in a string of regulatory approval that TEGSEDI has earned throughout Europe. It is supported by phase 3 data that demonstrates significant advantages in reducing symptoms of neuropathy and improving quality of life when compared to placebo. Now that this medication has been approved for reimbursement in Spain, adult patients living with polyneuropathy caused by hATTR amyloidosis will be able to experience a greater quality of life.