First Gene Therapy for ALS Approved: What Patients Should Know

May 16, 2023
Neil A. Shneider, MD, PhD

Neil A. Shneider, MD, PhD

The FDA has approved tofersen (Qalsody), the first drug developed for patients with a rare form of ALS caused by mutations in a gene called SOD1. ALS usually causes paralysis and death within a few years of diagnosis and is particularly aggressive in some patients with SOD1 mutations. 

In a phase 3 trial to test tofersen’s effectiveness, the drug did not significantly slow disease progression during the 28-week study period. However, the trial demonstrated that tofersen reduced levels of the toxic SOD1 protein and lowered levels of neurofilament light chain, a marker of neuronal injury.  

Based largely on these findings, tofersen was granted accelerated approval by the FDA. But to remain available, the FDA will require an additional study confirming clinical efficacy. 

Several patients have received tofersen at Columbia University’s Eleanor and Lou Gehrig ALS Center. We spoke with the center’s director, Neil Shneider, MD, PhD, about how patients have fared and where he thinks tofersen will have the biggest impact. [read more]

Source: CUIMC Newsroom