Tegsedi first Approved to treat stage 1 or 2 polyneuropathy in adults with FAP – also called transthyretin hereditary amyloidosis (hATTR) – by European Commission in 2018. According to Akcea Therapy, a subsidiary of Ionis Pharmacy which was originally developed by Tegsedi, this drug is the first of its kind approved for replacement in Italy, and the first can be given at home.
“Approval of reimbursement for TEGSEDI at Italy is an interesting milestone because people living with hATTR amyloidosis with polyneuropathy will now have much needed treatment options, and which can be given at home, “Michael Pollock, senior vice president, head of Europe at Akcea, said in a pers conference.
FAP is caused by an abnormal protein buildup transthyretin (TTR). Tegsedi is a modified RNA molecule called antisense oligonucleotides which can tie RNA messenger contains instructions for making TTR to reduce excess protein in organs throughout the body. This drug is given once a week by subcutaneous injection (under the skin).
Approval in Italy is based on data from the NEURO-TTR Phase 2/3 trial (NCT01737398), which tested the safety and effectiveness of Tegsedi in 173 adults with FAP for 15 months. Compared to placebo, Tegsedi significantly improved the quality of life of participants and reduced the development of neuropathic diseases.
“HATTR is a complex disease and we understand how important it is for patients to have access to treatment as soon as possible because this disease can quickly develop,” said Laura Obici, MD, trial researcher from Fondazione IRCCS Policlinico San Matteo.
“TEGSEDI is an important treatment choice for the hATTR community, and long-term data show that patients treated with TEGSEDI continue to experience an increase in neuropathy size and quality of life, with a greater effect seen when treatment begins earlier in the disease life cycle,” he add.
Ongoing extension studies (NCT02175004) is evaluating Tegsedi’s long-term safety and tolerability at a dose of 300 mg, once per week for up to five years, on 135 participants who completed the original NEURO-TTR trial. Results It has been shown that Tegsedi continues to improve the quality of life and facilitate the development of the disease in all participants, including those who have just started using Tegsedi during an extended trial (switching from placebo).
“We are pleased that after a detailed evaluation of TEGSEDI from the Italian Medicines Agency they recognized the serious unmet needs of hATTR amyloidosis patients in the country and the importance of safety and efficacy data shown so far from this therapy,” said Massimo Boriero, vice president, general manager of PT Italy in Akcea. “We look forward to continuing to work with the Italian Medicines Agency to quickly bring TEGSEDI to the right patients.”
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