Akcea Therapeutics, Inc. announced the Portuguese Medicine Regulatory Authority (INFARMED) has granted approval for reimbursement of TEGSEDI (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin (hATTR) amyloidosis. hATTR amyloidosis is an under-recognized, debilitating and progressive disease that is caused by the buildup of TTR proteins that misfold due to inherited genetic mutations. It is characterized by the deposition of amyloid fibrils throughout the body including in nervous tissue and can have a devastating impact on patients' quality of life. TEGSEDI is a once-weekly, at-home subcutaneous injection that targets the polyneuropathy of hATTR amyloidosis at its source by silencing the defective gene in these patients and reducing abnormal production of the TTR protein. It is the first antisense oligonucleotide medicine available for patients in Portugal with hATTR amyloidosis with polyneuropathy, and also the first antisense oligonucleotide treatment available to Portuguese patients that can be self-administered at home. INFARMED's positive opinion of reimbursement for TEGSEDI was based on results from the Phase 3 NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy. Results from that study demonstrated that patients treated with TEGSEDI experienced significant benefit compared to patients treated with placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression.