Tegsedi (Inotersen) approved by FDA for hATTR amyloidosis
The U.S. Food and Drug Administration today approved Tegsedi (inotersen), a novel therapy for the treatment of hereditary TTR amyloidosis polyneuropathy.
This approval comes after positive results from the Tegsedi clinical trial (NEURO-TTR) published in the New England Journal of Medicine in July 2018. The study met both of its primary endpoints and showed statistically significant benefit in both neuropathy impairment scores and quality of life measures compared to placebo.
The approval of Tegsedi comes just after the approval of the first treatment for hereditary amyloidosis. “The rapidly changing treatment landscape has drastically improved the outlook for those suffering from hereditary TTR amyloidosis,” says Dr. Morie Gertz, Mayo Clinic Hematology, Rochester, Minnesota. “The approval of Tegsedi is hugely important for hereditary TTR patients. It gives patients treatment options and hope where there very recently was not. The phase 3 clinical trial evidence supports the efficacy of this drug in improving clinical and quality of life measures compared to placebo. I am encouraged by the data and believe Tegsedi will lead to better outcomes for patients.”
Tegsedi is an RNA interference drug (RNAi) that reduces the production of the disease-causing TTR protein in the liver by blocking the activity of a gene. Tegsedi is administered once weekly as a single 300 mg subcutaneous injection.
Following FDA approval, Tegsedi can be prescribed for treatment of hereditary TTR amyloidosis polyneuropathy once the pharmaceutical company completes their preparations to bring the drug to market.
View more information about Tegsedi here>>
Read more about hereditary ATTR amyloidosis here>>
Read original press release from the FDA here>>