The FDA has given accelerated approval for QALSODY™ (tofersen) to treat SOD1-ALS, marking a significant scientific advancement as it is the first treatment to target a genetic cause of ALS.
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Biogen Inc. (Nasdaq – BIIB) has announced that the U.S. Food and Drug Administration (FDA) has approved QALSODY™ (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a specific genetic mutation.
This approval is based on a reduction in plasma neurofilament light chain (NFL) observed in patients who were treated with QALSODY, but further studies are needed to confirm its benefits. QALSODY is the first treatment approved to target a genetic cause of ALS.
Pain, exhaustion, arthralgia, an increase in white blood cells in the cerebrospinal fluid (CSF), and myalgia were the most frequently reported side effects.
A study was conducted to evaluate the effectiveness of QALSODY in treating patients with ALS who had a specific genetic mutation. The study was double-blind, placebo-controlled, and lasted for 28 weeks. A total of 108 patients aged between 23 and 78 years were randomly assigned to receive either QALSODY 100 mg (n=72) or placebo (n=36) for 24 weeks, with three loading doses followed by five maintenance doses. The confirmatory trial will be the ongoing Phase 3 ATLAS investigation of tofersen in patients with pre symptomatic SOD1-ALS.
The treatment will be made available in the U.S. to healthcare providers soon, but there may be variations in time to treatment as institutions and treatment centres learn about it.
About BioGen
Biogen Inc. is a multinational biotech company located in Cambridge, Massachusetts. They focus on developing and delivering therapies for neurological diseases to patients worldwide.
Date: April 25th 2023
Source: https://investors.biogen.com/news-releases/news-release-details/fda-grants-accelerated-approval-qalsodytm-tofersen-sod1-als
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