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Akcea announces approval for reimbursement of TEGSEDI® (inotersen) in Portugal for treatment of hereditary transthyretin amyloidosis with polyneuropathy
TEGSEDI is first antisense oligonucleotide medicine available to patients in Portugal for at-home subcutaneous injection Approval issued by Portuguese
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"TEGSEDI is first antisense oligonucleotide medicine available to patients in Portugal for at-home subcutaneous injection\n Approval issued by Portuguese Medicine Regulatory Authority only 14 months after submission, versus the average 21 months\n\n\nBOSTON, June 1, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today 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. \n\n \n \n \n \n \n \n\n \n\"We are very pleased that INFARMED was able to review and approve reimbursement of TEGSEDI quickly, making this important at-home treatment option available to people living with hATTR amyloidosis with polyneuropathy in Portugal,\" said Michael Pollock, SVP, head of Europe at Akcea. \"hATTR amyloidosis affects approximately 50,000 people worldwide, with Portugal having about 2,000 patients, a much higher incidence than most countries due to the prevalence of the V30M mutation. The approval is a testament to the strength of the efficacy and safety data supporting TEGSEDI, and the serious unmet need among patients living with hATTR amyloidosis in Portugal. We look forward to working with INFARMED to make TEGSEDI available to appropriate patients immediately.\" \nhATTR 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. \nTEGSEDI 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.\n\"TEGSEDI is an innovative therapy. Throughout the clinical development, TEGSEDI showed improve...