Date: 18-May-2020

Akceas Tegsedi Approved In Spain To Treat Polyneuropathy In Hereditary Transthyretin Amyloidosis

Akcea Therapeutics Inc, a biopharmaceutical company, announced The Ministry of Health of Spain (MSCBS) has granted approval for the reimbursement of Tegsedi (inotersen) in the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin (hATTR) amyloidosis.

"We appreciate the Ministry of Health's thorough evaluation of Tegsediand rapid approval to make this treatment option available to people living with hATTR amyloidosis with polyneuropathy in Spain," said Michael Pollock, SVP, head of Europe at Akcea.

"This milestone highlights the strength of the clinical data supporting Tegsediand the critical need in the hATTR amyloidosis community for a subcutaneous treatment option that allows patients to self-administer at a time and place that works for them. 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 Tegsediavailable to patients in Spain as soon as possible,”Michael added.

Hereditary Transthyretin (hATTR) amyloidosisis 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.

Tegsediis a once-weekly, at-home subcutaneous injection that targets the polyneuropathy of hATTR amyloidosis at its source by reducing production of the TTR protein. It is the first antisense medicine available for patients in Spain with hATTR amyloidosis with polyneuropathy, and also the first at-home treatment available to Spanish patients.

The Ministry of Health's positive opinion of reimbursement for Tegsediwas based on results from the phase 3 NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy. Results from that study demonstrated that patients treated with Tegsediexperienced significant benefit compared to patients treated with placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression.

Tegsedi (Inotersen) was approved by the US Food and Drug Administration (US FDA) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Tegsedi, discovered and developed by Ionis Pharmaceuticals, is the world's first and only subcutaneous RNA-targeting drug designed to reduce the production of human transthyretin (TTR) protein.

The approval is based on data from the NEURO-TTR study that was a phase 3 randomized (2:1), double-blind, placebo-controlled, 15-month, international study in 172 patients with hATTR amyloidosis with symptoms of polyneuropathy.

In NEURO-TTR, Tegsedidemonstrated significant improvement compared to placebo in measures of neuropathy and quality of life as measured by the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score. Patients treated with Tegsediexperienced similar benefit regardless of subgroups such as age, sex, race, region, Neuropathy Impairment Score (NIS), Val30Met mutation status, and disease stage.