Today, the FDA announced it has granted accelerated approval to tofersen, a treatment for people living with ALS tied to mutations of the SOD1 gene. This is a significant victory for the ALS community and our efforts to make ALS livable for everyone, everywhere, until we can cure it.  

It is the second time in less than a year that the FDA has approved a drug to treat ALS, and the third time in six years. 

Tofersen is an antisense oligonucleotide therapy, a small string of DNA letters that are designed to bind to specific molecules of RNA and stop the production of toxic proteins. Tofersen was developed to specifically target the RNA produced from a mutated SOD1 gene to stop toxic SOD1 proteins from being made.  

You can read our full statement on tofersen here.

While tofersen has been approved only to treat people living with SOD1-ALS, this is a win for the whole community.  

1. This marks the first use of accelerated approval in ALS. 
2. This was the first time in ALS that a biomarker was credibly used as a surrogate endpoint. 
3. The underlying technology behind tofersen is very promising. 
4. This is the first gene-based therapy for ALS to be approved. 
5. Tofersen is being studied to see if it can prevent the onset of ALS altogether. 
6. The entire ALS community was united in advocating for tofersen! 

As Larry Falivena, a member of The ALS Association’s board of trustees who has been living with ALS since 2017 and who participated in clinical trials of tofersen, said,

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“Every opportunity to make a new treatment available that can stop or slow down this disease is a win for the whole community." 

Listen to a discussion about the implications of tofersen’s approval with Dr. Kuldip Dave, The ALS Association’s vice president of research, here.  

To continue to follow stories about people living with ALS in the community and learn more about the disease, subscribe to receive our weekly blogs in your inbox HERE or follow us at als.org/blog. 

There are several FDA approved therapies available to treat ALS and its symptoms. Please consult your doctor or health care professional about which ones may be right for you.