The Portuguese Medicine Regulatory Authority, known as Infarmed, has added Tegsedi (inotersen) — an approved treatment for stage 1 or 2 polyneuropathy in adults with familial amyloid polyneuropathy (FAP) — to the list of reimbursed medications for patients living in Portugal.
Tegsedi now will be the first antisense medication that can be self-administered at home to become available at a lower cost to people in the country with FAP, also known as hereditary transthyretin (hATTR) amyloidosis. The move follows the medication’s recent approval for reimbursement in Spain.
“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,” Michael Pollock, senior vice president and head of Europe at Akcea Therapeutics, said in a press release.
“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,” Pollock added.
FAP is caused by the buildup of a misfolded protein called transthyretin (TTR) throughout the body. With around 2,000 patients, Portugal has a much higher incidence of the disease than other countries. This is due to the high prevalence of the FAP-causing Val30Met mutation.
Tegsedi is a chemically modified RNA molecule (antisense oligonucleotide) developed by Ionis Pharmaceuticals and its subsidiary Akcea to treat adults with FAP.
The medication is administered once per week through an under-the-skin injection that can be performed at home. It works by preventing cells from using the messenger RNA (mRNA) molecule that contains the instructions to make the TTR protein. By doing so, Tegsedi effectively lowers the production and buildup of TTR in organs and tissues throughout the body.
Infarmed’s decision to approve the reimbursement of Tegsedi was based on positive data from the Phase 2/3 NEURO-TTR trial (NCT01737398), which assessed the safety and efficacy of the medication in 173 adults with FAP for 15 months.
The results showed that, compared with a placebo, Tegsedi improved the participants’ quality of life, as assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy. It also eased neuropathic disease progression, as measured by the modified Neuropathy Impairment Score +7, compared with a placebo.
An extension study (NCT02175004), which enrolled 135 patients who completed the original NEURO-TTR trial, is currently underway to assess the long-term safety and tolerability of Tegsedi at a dose of 300 mg, once per week, for up to five years.
Data from the extension study released last year showed that Tegsedi continued to improve quality of life and slow disease progression in all patients, including those who only started treatment during the extension study.
“Throughout the clinical development, TEGSEDI showed improvement in quality of life and progression of neuropathy in patients with hATTR,” said Teresa Coelho, MD, coordinator of Corino de Andrade Unit of Centro Hospitalar Universitário of Porto.
“With this reimbursement approval, TEGSEDI is available for patients with hATTR and represents an important new treatment option for them,” Coelho said.
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