Tegsedi (inotersen), by Akcea Therapeutics and Ionis Pharmaceuticals, has been granted approval in Canada as a treatment for stage 1 or stage 2 polyneuropathy in adults with hereditary transthyretin (hATTR) amyloidosis, also called familial amyloid polyneuropathy (FAP).
This announcement comes at about the same time as Tegsedi’s approval for hATTR amyloidosis treatment by the U.S. Food and Drug Administration. Earlier this year, Tegsedi was also granted marketing authorization approval in Europe.
Health Canada had previously granted the therapy priority review, which helps fast-track the approval process for certain eligible new drug submissions.
“We appreciate Health Canada’s thorough and timely evaluation of TEGSEDI under priority review, and we look forward to working with all stakeholders to ensure that patients can receive timely and appropriate access to this important new treatment for Canadians living with hATTR amyloidosis,” Jared Rhines, general manager at Akcea Therapeutics Canada, said in a press release.
This now makes Tegsedi the first approved therapy for patients with hATTR amyloidosis in Canada.
hATTR results from the abnormal production of the mutated transthyretin (TTR) protein. Patients with hATTR amyloidosis have deposits of the mutated TTR protein in several tissues and organs within the body.
As these deposits progressively accumulate, patients develop sensory, motor, and autonomic (involuntary processes such as breathing) dysfunction, which negatively affects their quality of life.
Tegsedi is a once-a-week subcutaneous (under the skin) injection that works to reduce the production of TTR protein.
“We believe this achievement underscores Akcea’s commitment to the global rare disease community and our mission to deliver innovative therapies to patients no matter where they call home. We are excited to launch this innovative therapy along with Akcea ConnectTM, our comprehensive support program, for patients and their healthcare providers coast to coast,” Rhines said.
The decision to approve Tegsedi stemmed from positive results from the Phase 3 NEURO-TTR trial (NCT01737398), in which hATTR amyloidosis patients with symptoms of polyneuropathy were randomized in a 2:1 ratio to receive Tegsedi or a placebo.
The study was aimed at evaluating the effect of Tegsedi on two primary objectives — neurological function and quality of life. Neurological function was assessed using a test called the modified Neuropathy Impairment Score +7 (mNIS+7) and quality of life was measured using the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score.
Tegsedi was found to significantly improve both parameters for a large percentage of patients in the study.
“hATTR amyloidosis is a debilitating disease that carries with it significant morbidity and mortality, and we are extremely limited in options to offer patients with this disease and their families. The approval of inotersen changes this and is a welcome advance. In NEURO TTR, the study upon which Health Canada approval was sought, the changes from baseline showed statistically significant benefit in favor of inotersen treatment, including polyneuropathy symptoms and quality of life,” said Vera Bril, MD, professor of medicine at the University of Toronto and director of neurology at Mount Sinai Hospital.
Detailed results from the trial were published in a study titled, “Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis,” in the New England Journal of Medicine.
“Today marks an exciting day for Canadians living with hATTR amyloidosis and their families as inotersen [Tegsedi] is the first approved disease-modifying therapy to address the significant burden many bear in living with debilitating and progressive symptoms,” said Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders. “A tremendous emotional burden often comes with an inherited disease, and we believe this approval, and the innovation behind this treatment, means that today an important step in their hope for a brighter future has been realized. We now look forward to Akcea working with stakeholders to make this drug available to patients as soon as possible.”