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Positive CHMP opinion for Akcea orphan drug inotersen

Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending approval of its orphan drug inotersen (Tegsedi), for the treatment of Stage 1 or Stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis (hATTR amyloidosis).1

hTTAR amyloidosis is a fatal hereditary disease caused by abnormal formation and build-up of TTR amyloid deposits in the body,2 leading to debilitating symptoms, such as loss of sensation in the limbs, chest pain, palpitations, weight loss, nausea, weakness, fatigue and chest pain.3 People with hATTR amyloidosis die within three to fifteen years of developing symptoms.4

Inotersen aims to affect the course of the disease and improve quality of life.5

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The positive opinion is based on results from the Phase 3 NEURO-TTR study and Open Label Extension (OLE) study in patients with hATTR amyloidosis with symptoms of polyneuropathy.6 The results showed that inotersen resulted in significant benefits for patients, compared with placebo on both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy score, and the modified Neuropathy Impairment Score + 7, a measure of neuropathic disease progression.6

“We are delighted at this positive opinion, which is an important milestone for people suffering with this disease. Patients currently have no treatment options and the prognosis is potentially devastating,” says Luke Robinson, General Manager, Akcea Therapeutics, UK, Ireland & Nordics. “This encouraging announcement from CHMP offers hope to these patients and their families, who may soon have access to an innovative therapy that can help improve their lives.”


1 European Medicines Agency. Summary of opinion (initial authorisation) Tegsedi. Available at: Last accessed June 2018.
2 Gertz MA, et al. (2015) Diagnosis, Prognosis, and Therapy of Transthyretin Amyloidosis. JACC. 66(21):2451-66.
3 Amyloidosis Patient Information Site. FAQs. Available at: Last accessed June 2018.
4 Gertz MA. (2017) Hereditary ATTR Amyloidosis: Burden of Illness and Diagnostic Challenges. AJMC. (7 Suppl):S107-S112.
5 European Medicines Agency. (2018) New medicine for hereditary rare disease. Tegsedi addresses unmet medical need for treatment of hereditary transthyretin amyloidosis. Available at: Last accessed June 2018.
6 Gertz MA. (2018) Safety and Efficacy of Inotersen in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (NEURO-TTR) (N2.001). Neurology. 90 (15 Suppl