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Akcea and Ionis Announce European Commission Approval of Tegsedi™ ▼ (inotersen)

16 Jul 18

Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that TegsediTM (inotersen) has received marketing authorization approval from the European Commission (EC) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). This follows the positive opinion recommending approval provided by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).[i] Click here to view the EC’s decision.

“With the EC’s decision, inotersen is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis. With subcutaneous delivery, inotersen puts treatment in the patients’ hands while bringing the significant benefits shown in the NEURO-TTR study in both measures of neuropathy and quality of life for people living with this serious and fatal disease. This is an important day for the hATTR amyloidosis community as we believe inotersen enables people and their families impacted by this disease to move forward with their lives,” said Paula Soteropoulos, chief executive officer at Akcea Therapeutics. “Today is a milestone for Akcea with our first drug approval. It is an achievement we share with the courageous hATTR patient community in Europe and around the globe. We are ready to launch inotersen along with our patient and physician support services across Europe.” 

The abnormal formation and aggregation of transthyretin (TTR) protein results in TTR amyloid deposits throughout the body and is the underlying cause of hATTR amyloidosis. Inotersen is designed to block production of the TTR protein. In the NEURO-TTR study, treatment with inotersen produced substantial reductions in the levels of the TTR protein regardless of mutation type or stage of disease.2

"hATTR amyloidosis is an inherited, progressive and fatal disease for which treatment options are limited. The approval of inotersen brings us into a new era of treatment with an efficacious and disease modifying medicine that potentially allows patients to achieve a greater degree of independence,” said Teresa Coelho, M.D., neurologist and neurophysiologist at Santo António Hospital, Porto, Portugal. “Inotersen has demonstrated rapid and sustained benefits in improving the course of this disease and preserving quality of life.”

The European Commission’s approval of inotersen was based on results from the Phase 3 NEURO-TTR study in patients with hATTR amyloidosis, with symptoms of polyneuropathy.[ii] Results from that study demonstrated that patients treated with inotersen experienced 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<

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