The FDA approved Akcea Therapeutics’ Tegsedi (inotersen) for the treatment of polyneuropathy caused by hereditary transthyretin (hATTR)-mediated amyloidosis in adults. The self-administered RNA-targeting therapeutic was approved by European agencies three months ago.
Tegsedi’s approval has come shorty after the FDA clearance of Alnylam Pharmaceuticals’ Onpattro (patisiran) for the treatment of adults with polyneuropathy due to hATTR, marking the first drug authorized for this indication in the U.S.
While Akcea has currently priced Tegsedi at a similar level to Onpattro (around $345,000 to $450,000 a year), Akcea CEO Paul Soteropoulos commented that the company is considering strategies to increase affordability. She noted that “its self-administration gives the flexibility to treat at a time that works for [patients], which could change the way this progressive and debilitating disease is treated and managed.” Onpattro is administered via infusion at a clinic once every three weeks.
Despite being self-administered, the FDA required Tegsedi to carry carry boxed warnings related to thrombocytopenia and glomerulonephritis, with patients receiving the therapy requiring enhanced monitoring, including weekly checks of platelet counts, kidney function every two weeks and liver function every four months.
Approval of Tegsedi was based on results from the Phase III NEURO-TTR study. The findings demonstrated that patients treated with Akcea’s drug experienced significant benefit compared to patients treated with placebo across both co-primary endpoints. Earlier this year, the FDA extended its review of the filing for Tegsedi by three months to allow it more time to assess additional data.