Inotersen

WAINUA™ (eplontersen) Granted First-Ever Regulatory Approval in the US for the Treatment of Adults With Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

Retrieved on: 
Friday, December 22, 2023
Research, FDA, Neurology, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, Science, Quality of life, Tissue, Heart, Heredity, Serum, Polyneuropathy, Death, Inotersen, Ageing, TTR, JAMA, Vitamin A, Diagnosis, Metabolism, Amyloidosis, Pancreas, Disease, Safety, Journal, Heart failure, Disability studies, Sympathetic nervous system, Vitamin A deficiency, Food, Biopharmaceutical, AstraZeneca, Caregiver, ATTR, Patient, Medicine, LICA, Ionis, American Medical Association, Growth, Central nervous system, Pharmaceutical industry

It is also a pivotal moment for Ionis as WAINUA will be the first in a steady cadence of potential commercial launches for the company.

Key Points: 
  • It is also a pivotal moment for Ionis as WAINUA will be the first in a steady cadence of potential commercial launches for the company.
  • Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels.
  • Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.
  • Please see link to US Full Prescribing Information for WAINUA.

Eplontersen demonstrated sustained benefit in Phase III trial for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) through 66 weeks

Retrieved on: 
Monday, March 27, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Disability studies, Tissue, Heart, Sensory loss, US Foods, Central nervous system, University, CV, Heart failure, Metabolism, Serum, Diabetic neuropathy, Heredity, Pancreas, Ageing, Quality of life, American Academy, Department of Neurology, Xuanwu hospital, Biopharmaceutical, AstraZeneca, Central nervous system disease, Sympathetic nervous system, LICA, Inotersen, Penn Presbyterian Medical Center, Ionis, Muscle weakness, TTR, ATTR, Patient, Orphan, Cardiomyopathy, Diagnosis, Growth, Phase, Polyneuropathy, Death, Safety, Perelman School of Medicine at the University of Pennsylvania, Disease, Medical imaging, Pharmaceutical industry, Science, Neurology

Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed eplontersen met its co-primary endpoints through 66 weeks.

Key Points: 
  • Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed eplontersen met its co-primary endpoints through 66 weeks.
  • The trial also met its third co-primary endpoint demonstrating a statistically significant reduction in serum TTR concentration versus an external placebo group.
  • Eplontersen continued to demonstrate a safety and tolerability profile consistent with that observed at 35 weeks.
  • We are encouraged by the sustained benefit demonstrated by eplontersen and what a self-administered treatment could mean for patients and families affected by ATTRv-PN.

Akcea Announces Approval for Reimbursement of TEGSEDI® (inotersen) in Austria for Treatment of Hereditary Transthyretin Amyloidosis with Polyneuropathy

Retrieved on: 
Wednesday, July 22, 2020
Organ systems, Branches of biology, Neurological disorders, Proteins, Orphan drugs, Protein folding, RTT, Familial amyloid polyneuropathy, Amyloidosis, Transthyretin, Inotersen, Polyneuropathy

"Research also suggests that earlier access to treatment for hATTR amyloidosis may help slow the progression of the disease, making this approval of reimbursement even more significant for the hATTR amyloidosis community in Austria."

Key Points: 
  • "Research also suggests that earlier access to treatment for hATTR amyloidosis may help slow the progression of the disease, making this approval of reimbursement even more significant for the hATTR amyloidosis community in Austria."
  • Since then, TEGSEDI has continued to receive regulatory and reimbursement approvals globally, most recently gaining reimbursement approvals in Italy and Portugal.
  • Food and Drug Administration(FDA) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
  • TEGSEDI also received marketing authorization in theEuropean Unionand Canada for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis.