WebbBiogen is seeking approval of tofersen, an antisense oligonucleotide (ASO) drug, through the FDA's Accelerated Approval Program. According to the FDA, this program was instituted “to allow for earlier approval of drugs that treat serious conditions, and fill an unmet medical need based on a surrogate endpoint.” Webb11 apr. 2024 · Federal regulators are considering the approval of the drug tofersen to treat a rare genetic form of amyotrophic lateral sclerosis (ALS).; This version affects about 2% of people around the world ...
BIIB067 (Tofersen) MND Association
Webb18 okt. 2024 · Amylyx Pharmaceuticals, a Boston-area biotech working on another closely watched ALS drug, recently announced the agency agreed to allow it to submit its drug for approval before conducting an additional late-stage study. Amylyx's drug, known as AMX0035, succeeded in a late-stage clinical trial last year. Patients who received it … Webb17 okt. 2024 · Today, the company has a broad pipeline of investigational drugs being evaluated in ALS. About Tofersen Tofersen is an antisense drug being evaluated for the … free cad software 2023
Tofersen Safe, Appears to Slow Disease Progression in SOD1 ALS...
Webb23 mars 2024 · If approved, tofersen will be the first treatment targeting a genetic cause of ALS and the next marketed Ionis-discovered antisense medicine ; FDA decision expected by April 25, 2024; CARLSBAD, Calif., March 22, 2024 /PRNewswire/ -- Ionis (Nasdaq: IONS) today announced the outcome of the U.S. Food and Drug Administration's (FDA) … Webb24 mars 2024 · “If approved, tofersen would potentially represent a major advance for people living with SOD1-ALS.” Considering NfL as a good surrogate biomarker of efficacy … Webb17 okt. 2024 · While tofersen did not meet the primary endpoint of change from baseline to week 28 in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS … free cad software app for iphone