Release Details
Tegsedi® Receives Innovative Drug Category Pricing in Brazil
Tegsedi has been approved by the Brazilian regulatory agency ANVISA (Agência Nacional de Vigilância Sanitária) for the treatment of Stage 1 or 2 polyneuropathy (nerve damage) in adult patients with hereditary transthyretin-mediated (hATTR) amyloidosis to delay disease progression and improve quality of life. It is the first antisense medicine available for patients in
"This is an incredibly important decision in allowing patients access to Tegsedi in
Studies have proven that Tegsedi significantly improved measures of neuropathic disease progression and quality of life for patients.1 There are estimated to be 5,000 patients with hATTR amyloidosis in
About Tegsedi® (inotersen)
Tegsedi is designed to block the production of the TTR protein, which is the underlying cause of hATTR amyloidosis. It is a novel, first-in-class 2'-O-2- methoxyethyl phosphorothioate antisense oligonucleotide and its mechanism of action is distinct from all previous therapies for hATTR amyloidosis. In the Phase 3 NEURO-TTR study, Tegsedi significantly reduced the levels of TTR protein in patients irrespective of mutation type or stage of disease,1 and significantly improved quality of life.1 Tegsedi is a once-weekly, self-administered subcutaneous treatment, which enables greater patient independence. Tegsedi is approved in
Tegsedi, a product of Ionis Pharmaceutical's proprietary antisense technology, is an antisense medicine designed to reduce the production of transthyretin (TTR) protein.
About Hereditary Transthyretin (hATTR) Amyloidosis
hATTR amyloidosis is a progressive, systemic and fatal inherited disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone marrow. The progressive accumulation of TTR amyloid deposits in these tissues and organs leads to sensory, motor and autonomic dysfunction often having debilitating effects on multiple aspects of a patient's life. Patients with hATTR amyloidosis often present with a mixed phenotype and experience overlapping symptoms of polyneuropathy and cardiomyopathy.
Ultimately, hATTR amyloidosis results in death within three to 15 years of symptom onset. Therapeutic options for the treatment of patients with hATTR amyloidosis are limited and there are currently no disease-modifying drugs approved for the disease. There are an estimated 50,000 patients with hATTR amyloidosis worldwide. Additional information on hATTR amyloidosis, including a full list of organizations supporting the hATTR amyloidosis community worldwide, is available at www.hattrchangethecourse.com.
About
PTC is a science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us on www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.
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As with any pharmaceutical under development, there are significant risks in the development, regulatory approval, and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Tegsedi.
The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.
References
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1 Benson MD,et al. Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018; 379:22-3.
2 Schmidt H,
3 Gertz MA. Hereditary ATTR Amyloidosis: Burden of Illness and Diagnostic Challenges. Am J Manag Care. 2017;23(7 suppl):S107-S112.
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