First Gene-Specific ALS Treatment Approved on DNA Day

Brook Croke

April 25, 2023

Brook Croke
February 9, 2024

Today is a wonderful day for the ALS community. Today, the first gene-specific therapy for ALS, QALSODY, formerly called tofersen, was granted accelerated approval by the US Food and Drug Administration. Biogen collaborated with Ionis Pharmaceuticals on the early development of QALSODY, an antisense oligonucleotide (ASO) therapy. Individuals with SOD1-ALS are now eligible for the medication which is administered through lumbar puncture.

Variants in the SOD1 (superoxide dimutase 1) gene were first discovered to be associated with an increased risk for ALS in 1993. Thirty years later, the first gene-specific treatment is available and this is definitely something to celebrate! It does not seem like a coincidence that approval comes on DNA Day, the 70th anniversary of the discovery of the DNA double helix and the 20th anniversary of the completion of the Human Genome Project.

But, even as we celebrate the FDA’s decision and this drug’s efficacy, we also remember the patients and caregivers who were unable to access the drug or who passed away without witnessing this momentous occasion. Two patients, in particular, are especially close to our Synapticure team’s hearts: John and Ethan Andrews, sons of our care coordinator, Rebecca, participated in the VALOR study, a clinical trial that generated data the FDA required for approval of QALSODY. We and the entire ALS community are so grateful for individuals like John and Ethan who committed to participating in research for the good of the community. We remember John and Ethan, who are no longer with us, and thank all of the individuals that participated in the VALOR phase III trial—many in the midst of a global pandemic—to make this approval possible. 

Questions remain regarding costs and insurance coverage of the medication as well as when access will roll out across the US. Currently, individuals with SOD1-ALS are able to continue to receive the life-saving medication through clinical trial sites enrolling individuals through the expanded access program. Presymptomatic individuals with specific SOD1 variants are eligible for the ATLAS trial that will continue to monitor effects of this medication in individuals early in the ALS disease process.

As gene-specific therapies continue to be developed and approved, the importance of offering genetic testing and counseling continues to grow in importance. Don’t hesitate to reach out to our team if you or someone you know could benefit from genetic testing, which is available at no cost to individuals with ALS and those with a family history of ALS. If you are interested in learning more about Synapticure and the services we provide, please sign up to learn more.

As new information becomes available, we will update this blog post and share, so stay tuned…

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