Pharma Focus Asia

Akcea and Ionis Announce Approval of TEGSEDI ™ (Inotensive Injection) in Canada

Monday, October 08, 2018

Akcea Therapeutics, Inc., a company affiliated with Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced the approval of TEGSEDI ™ ( Inotersen injection) in Canada for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin-mediated (hATTR) amyloidosis.

Following an accelerated regulatory process by Health Canada, TEGSEDI, a drug with RNA interference, is the first approved treatment for Canadians suffering from hATTR amyloidosis. This disease is caused by abnormal formation of transthyretin (TTR protein), which leads to deposition of abnormal amyloid proteins in various body organs and tissues. The progressive deposition of these proteins leads to sensory, motor and vegetative dysfunctions, which in many ways affect the patient's life.

"We appreciate the thorough and timely evaluation of TEGSEDI as part of an accelerated approval process by Health Canada and look forward to working with all stakeholders to ensure that patients in Canada with hATTR amyloidosis have timely and adequate access to this new Treatment, "said Jared Rhines, general manager of Akcea Therapeutics Canada. "Following the marketing approval for TEGSEDI in Europe, this announcement is also an exciting milestone for Akcea due to our first drug approval in Canada. We believe this success underscores Akcea's commitment to treating rare disease patients around the world and our mission to develop innovative therapies for patients around the world. We're glad,

TEGSEDI is a subcutaneous injection that patients can self-administer once a week and that fights the origin of hATTR amyloidosis by reducing the production of TTR protein.

"Today is an exciting milestone for Canadians suffering from hATTR amyloidosis and their families, as Inotersen is the first approved disease-modifying therapy to combat the significant burden that many suffer with restrictive and progressive symptoms. A hereditary disease is often accompanied by an immense emotional burden. In our opinion, this authorization and the innovation associated with this treatment mean that today an important step can be taken in the hope of a better future. We look forward to working with Akcea with stakeholders to make this drug available to patients as quickly as possible, "said Durhane Wong-Rieger.

"HATTR amyloidosis is an incurable hereditary disease associated with significant morbidity and mortality. So far, the options we have been able to provide to patients and their families have been very limited. The approval of inotersen is changing that and is a welcome advance. In the NEURO TTR trial, which was targeted for approval by Health Canada, the changes from baseline indicated statistically significant benefits of inot- ser treatment, such as the symptoms of polyneuropathy and quality of life, "said Drs. Vera Bril, University of Toronto Professor of Medicine, University Health Network's Director of Neurology & Mount Sinai Hospital and Krembil Family Chair in Neurology.

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