FDA approves joint development of antisense oligonucleotide drug Tegsedi (inotersen) to go on the market

hATTR is a deadly performing genetic disease caused by abnormal folding of transthyroxineprotein (TTR) in the patient, which causes amyloid TTR to be deposited in various tissues and organs of the bodyrecently, Io
nis Pharmaceuticals and itscompany,, jointly announced that the U.SFDA(inotersen) approved the joint development of the anti-emily nucleotidedrug(inotersen) for the treatment of hereditary thyroxine amyloid degeneration (hATTR) patients with polyneuropathy (polyneuroneuro)TegsediTegsedi is an anti-human TTR
synthetic(ananifiable oligonucleotide drug, which, in combination with mRNA encoded with TTR protein, can lead to degradation of mRNA, thereby reducing the level of TTR protein (wild and mutant)Tegsedi is a weekly subcutaneous injection that allows patients to self-incur at home, Tegsedi has been approved in the United States, the European Union and Canadarelated studies
this approval is based on Tegsedi's performance in a randomized, double-blind, placebo-controlled International Phase 3 Clinical Trial called NEURO-TTR In this clinical study, 172 hATTR patients who showed symptoms of multiple neuropathy were treated with Tegsedi or placebo at a 2:1 ratio The researchers used the mNIS-7 and Norfolk QOL-DN scales to test for patients' neurological function and quality of life results showed that Tegsedi reached the common primary endpoint of the trial, significantly improving the patient's mNIS plus 7 and Norfolk QOL-DN scores At the same time, Tegsedi therapy significantly reduced TTR levels in patients, reducing levels of TTR protein in serum by an average of 79% regardless of what genetic mutation they were carrying or at what stage of disease development