Akcea, Ionis get FDA nod for Tegsedi
Akcea Therapeutics and Ionis Pharmaceuticals have received clearance from the Food and Drug Administration for Tegsedi (inotersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated, or hATTR-mediated, amyloidosis in adults.
"Tegsedi is the first and only RNA-targeting therapeutic that powerfully reduces the production of TTR protein through a once-weekly subcutaneous injection offering patients an effective treatment for people living with polyneuropathy caused by hATTR amyloidosis. We believe this profile will make Tegsedi an excellent choice for many patients and that it’s self-administration gives the flexibility to treat at a time that works for them which could change the way this progressive and debilitating disease is treated and managed,” Akcea Therapeutics CEO Paula Soteropoulos said, in a statement.
In hATTR amyloidosis, transthyretin, or TTR, protein misfolds and accumulates as amyloid deposits throughout the body. Tegsedi targets the disease at its source by reducing the production of TTR protein, according to the company.
"Tegsedi is the first and only RNA-targeting therapeutic that powerfully reduces the production of TTR protein through a once-weekly subcutaneous injection offering patients an effective treatment for people living with polyneuropathy caused by hATTR amyloidosis. We believe this profile will make Tegsedi an excellent choice for many patients and that it’s self-administration gives the flexibility to treat at a time that works for them which could change the way this progressive and debilitating disease is treated and managed,” Akcea Therapeutics CEO Paula Soteropoulos said, in a statement.
In hATTR amyloidosis, transthyretin, or TTR, protein misfolds and accumulates as amyloid deposits throughout the body. Tegsedi targets the disease at its source by reducing the production of TTR protein, according to the company.