FDA OKs Treatment for Polyneuropathy in Hereditary Transthyretin-Mediated Amyloidosis


Inotersen (Tegsedi) once-weekly subcutaneous injection is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis.

Officials with the FDA have approved inotersen (Tegsedi) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults, according to a press release.

Inotersen is the first FDA-approved RNA-targeting therapeutic that reduces production of the transthyretin (TTR) protein through a once-weekly subcutaneous injection.

“We believe this profile will make Tegsedi an excellent choice for many patients and that it’s self-administration gives flexibility to treat at a time that works for them which could change the way this progressive and debilitating disease is treated and managed,” Paula Soteropoulos, chief executive officer at Akcea Therapeutics, said in a press release.

The approval is based on data from the phase 3 NEURO-TTR study, which evaluated patients with hATTR amyloidosis with symptoms of polyneuropathy. According to the results, patients treated with inotersen experienced significant benefit compared with patients treated with a placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy and modified Neuropathy Impairment Score +7, a measure of neuropathic disease progression.

“hATTR amyloidosis is a fatal disease that affects multiple organs and body systems and robs people of so much—the ability to work, the ability to carry out daily tasks—all the things that make them feel in control of their lives,” Morie Gertz, MD, hematologists and chair emeritus of Internal Medicine at Mayo Clinic, said in the release. “Tegsedi has demonstrated nearly 80% reduction in TTR, which is now a validated approach to treating this disease.”

The treatment is associated with risk of thrombocytopenia and glomerulonephritis, according to the press release. Patients receiving inotersen should be closely monitored to support early detection and management of these identified risks.

Additionally, Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, announced they are working together with Accredo Specialty Pharmacy, a subsidiary of Express Scripts, to distribute inotersen.

“Tegsedi is a weekly self-administered injection, that should reduce administration costs and drug wastage,” Steve Miller, MD, chief medical officer at Express Scripts, said in a statement. “By introducing competition into the marketplace this should drive down the total cost of care.”


Akcea and Ionis Receive FDA Approval of TEGSEDI™ (inotersen) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults [news release]. Ionis’ website. http://ir.ionispharma.com/news-releases/news-release-details/akcea-and-ionis-receive-fda-approval-tegseditm-inotersen. Accessed October 8, 2018.

Akcea Announces Its Access and Distribution Strategy for TEGSEDI™ (inotersen) [news release]. Ionis’ website. http://ir.ionispharma.com/news-releases/news-release-details/akcea-announces-its-access-and-distribution-strategy-tegseditm. Accessed October 8, 2018.

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