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Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California, that specializes in discovering and developing RNA-targeted therapeutics.The company has three commercially approved medicines: Spinraza (), Tegsedi (), and Waylivra (Volanesorsen), and has four drugs in pivotal studies: tominersen for Huntington's disease (together with Roche), tofersen for SOD1-ALS, AKCEA ...
Ionis CEO Brett Monia said the company estimates that only 20% of the patients suffering from ATTR-PN are on treatment. "This is a market where more than one player can have great success," he added.
Tofersen was developed by Ionis Pharmaceuticals and was licensed to, and co-developed by, Biogen. [7] [8]The effectiveness of tofersen was evaluated in a 28-week, randomized, double-blind, placebo-controlled clinical study in 147 participants with weakness attributable to amyotrophic lateral sclerosis and a superoxide dismutase 1 (SOD-1) mutation confirmed by a central laboratory. [2]
IONIS-GCCR Rx, also known as ISIS-426115, is an antiglucocorticoid which is under development by Ionis Pharmaceuticals (formerly Isis Pharmaceuticals) for the treatment of diabetes mellitus type 2. [ 1 ] [ 2 ] It has also been under investigation for the treatment of Cushing's syndrome , but no development has been reported. [ 1 ]
The company has reached an agreement with the FDA on the requirements for accelerated approval and plans to submit a biologics license application in early 2025.
IONIS School of Technology and Management; Ionis Pharmaceuticals This page was last edited on 30 September 2018, at 19:02 (UTC). Text is available under the ...
In November 2016, the new drug application was accepted under the FDA's priority review process on the strength of the Phase III trial and the unmet need, and was also accepted for review at the European Medicines Agency (EMA) at that time. [21] [22] It was approved by the FDA in December 2016 and by EMA in May 2017 as the first drug to treat SMA.
This post is part of a series where personal finance expert Dan Solin provides 10 insurance tips no one else will tell you. See all 10, plus one bonus tip!Ratings are often bandied about as the ...