Tegsedi Available to FAP Patients in Spain at Reduced Cost

Tegsedi Available to FAP Patients in Spain at Reduced Cost
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Spain’s Ministry of Health has added Tegsedi (inotersen), an injection  treatment for stage 1 or 2 polyneuropathy in adults with familial amyloid polyneuropathy (FAP), to the national list of prescription medicines that patients are reimbursed for using.

With this decision, Tegsedi will be the first at-home antisense medication available to FAP patients in Spain under a reimbursement program that makes it available at a relatively low cost.

“We appreciate the Ministry of Health’s thorough evaluation of Tegsedi … to make this treatment option available to people living with hATTR amyloidosis with polyneuropathy in Spain,” Michael Pollock, a senior vice president and head of Europe at Akcea Therapeutics, said in a press release.

“This milestone highlights the strength of the clinical data supporting Tegsedi and the critical need in the hATTR amyloidosis community for a subcutaneous [under the skin] treatment option that allows patients to self-administer at a time and place that works for them,” Pollock added.

FAP, also known as hereditary transthyretin (hATTR) amyloidosis, is characterized by the buildup of a misfolded protein called transthyretin (TTR) in different tissues and organs.

Tegsedi, marketed by Ionis Pharmaceuticals and its subsidiary Akcea, is a chemically modified RNA molecule (antisense oligonucleotide) designed to bind to the messenger RNA (mRNA) molecule that provides cells with instructions to make the TTR protein.

Once bound to TTR’s mRNA, Tegsedi prevents cells from using the molecule, effectively reducing protein production in the liver — the organ that makes most of this protein.

The medication is administered once-a-week through an under the skin injection that can be performed at home. It was the first RNA-targeted therapy to be approved for the treatment of adults with FAP in the U.S., European Union, and Canada.

The ministry’s decision to approve the reimbursement of Tegsedi came six months after a request was submitted. It was based on positive data from the Phase 2/3 NEURO-TTR trial (NCT01737398).

Results showed that when given over 15 months, Tegsedi improved quality of life (assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy) and reduced neuropathic disease progression (assessed by the modified Neuropathy Impairment Score +7) in patients with FAP and polyneuropathy compared with a placebo.

An extension study (NCT02175004) in 135 patients who completed the NEURO-TTR trial is underway, with all being treated with Tegsedi, 300 mg once a week, for up to five years.

Findings from this extension trial, released in 2019, showed that Tegsedi continued to improve quality of life and slow disease progression. This was true for patients who had been receiving the medication for more than two years — 15 months in NEURO-TTR plus 12 months in the extension study — and for those who switched from a placebo in the original trial to Tegsedi in the extension study.

“The data from the NEURO-TTR study showing benefits in measures of neuropathy and quality of life underlines the potential of this therapy to benefit patients in the years ahead. We will continue to collaborate with the Ministry of Health to make Tegsedi available to patients in Spain as soon as possible,” Pollock said.

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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José holds a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.

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Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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