FAP Treatment Tegsedi Approved for Reimbursement in Italy

The Italian Medicines Agency has added Tegsedi (inotersen), a treatment for familial amyloid polyneuropathy (FAP), to the list of reimbursed therapies in the country.

Tegsedi was approved to treat stage 1 or 2 polyneuropathy in adults with FAP — also called hereditary transthyretin (hATTR) amyloidosis — by the European Commission in 2018. According to Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals which originally developed Tegsedi, the medication is the first of its kind to be approved for reimbursement in Italy, and the first that can be administered at home.

“The approval of reimbursement for TEGSEDI in Italy is an exciting milestone as people living with hATTR amyloidosis with polyneuropathy will now have a much-needed treatment option available, and one that can be administered at home,” Michael Pollock, senior vice president, head of Europe at Akcea, said in a press release.

FAP is caused by an abnormal buildup of the protein transthyretin (TTR). Tegsedi is a modified RNA molecule called antisense oligonucleotide that can bind to the messenger RNA containing instructions to make TTR to lower excess protein in organs throughout the body. The medication is administered once per week by subcutaneous (under-the-skin) injection.

The approval in Italy was based on data from the NEURO-TTR Phase 2/3 trial (NCT01737398), which tested Tegsedi’s safety and effectiveness in 173 adults with FAP for 15 months. Compared to a placebo, Tegsedi significantly improved participants’ quality of life and eased neuropathic disease progression.

“hATTR is a complex disease and we understand how important it is for patients to have access to treatment as soon as possible as the disease can quickly progress,” said Laura Obici, MD, trial investigator from Fondazione IRCCS Policlinico San Matteo.

“TEGSEDI is an important treatment option for the hATTR community, and long-term data shows that patients treated with TEGSEDI continue to experience improvements in measures of neuropathy and quality of life, with greater effects seen when treatment is started earlier in the disease lifecycle,” she added.

An ongoing extension study (NCT02175004) is evaluating the long-term safety and tolerability of Tegsedi at a dose of 300 mg, once per week for up to five years, in 135 participants who completed the original NEURO-TTR trial. Results have shown that Tegsedi continued to improve quality of life and ease disease progression in all participants, including those who only started on Tegsedi during the extension trial (switched from placebo).

“We are pleased that following the Italian Medicines Agency’s detailed evaluation of TEGSEDI they are recognizing the serious unmet need of hATTR amyloidosis patients in the Country and the significance of the safety and efficacy data demonstrated thus far from this therapy,” said Massimo Boriero, vice president, general manager of Italy at Akcea. “We look forward to continue to work with the Italian Medicines Agency to rapidly bring TEGSEDI to appropriate patients.”